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  • 16-001: Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents. - NCT03020030

    To test a novel risk group classification scheme with the goal of reducing rates of treatment-related toxicity without compromising complete remission rates and overall event-free survival. a. To test whether remission induction can be de-intensified in low-risk B-ALL patients without adversely impacting complete remission rates or proportion of patients classified as very high risk on the basis of high minimal residual disease (MRD levels). b. To determine rates of event-free survival, disease-free survival and overall survival associated with novel risk group classification scheme.

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    • Protocol Number:
      111805

    • Principal Investigator:
      Peter Cole M.D.

    • Phase:
      Phase III

    • Scope:
      National

    • Applicable Disease Sites:
      Leukemia, not otherwise specified,Lymphoid Leukemia,Leukemia, other

      • Contacts:

      • Rutgers University Prinicipal Investigator: Peter Cole M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

      1. Confirmed diagnosis of acute lymphoblastic leukemia. Diagnosis should be made by bone marrow aspirate or biopsy demonstrating ≥ 25% involvement by lymphoblasts, with flow cytometry or immunohistochemistry confirming B-precursor or T-ALL phenotype.
    • - For patients with circulating blasts in the peripheral blood, flow cytometry confirmation of B-ALL or T-ALL phenotype is sufficient for registration onto the study. Bone marrow aspirate and/or biopsy should be performed as soon as feasible, preferably prior to the initiation of any therapy. 2. Prior Therapy: No prior therapy is allowed except for the following:
    • Corticosteroids: Short courses of corticosteroid (defined as ≤ 7 days of corticosteroids within the 4-weeks preceding registration) are allowed prior to registration.
    • -- Participants who have been on corticosteroids chronically (defined as more than 7 days of corticosteroids within the 4-weeks preceding registration or more than 28 days of corticosteroids over the preceding 6 months) are not eligible.
    • IT cytarabine: A single dose of intrathecal cytarabine (at the time of the diagnostic lumbar puncture) is allowed prior to registration. If patient has received IT cytarabine prior to registration, Day 1 IT cytarabine should not be administered.
    • Emergent Radiation Therapy: Emergent radiation to the mediastinum or other life-threatening masses is allowed prior to registration. 3. Age: 365 days to < 22 years 4. Direct bilirubin < 1.4 mg/dL (23.9 micromoles/L). 5. Ability of parent or guardian to understand and the willingness to sign a written informed consent document.

    Exclusion Criteria:

      1. Mature B-cell (Burkitt's) ALL (defined by the presence of surface immunoglobulin and/or the t(8;14)(q24;q32), t(8;22), or t(2;8) translocation and/or c-myc-gene rearrangement). 2. World Health Organization diagnostic criteria of mixed phenotype acute leukemia (MPAL) or leukemia of ambiguous lineage 3. Any chemotherapy or radiotherapy for previous malignancy are not eligible. 4. Treatment in past with any anti-neoplastic agent, including methotrexate, 6-mercaptopurine, 6-thioguanine, vincristine cyclophosphamide, cytarabine (except for IT cytarabine), or any anthracycline, for any reason (eg, rheumatologic or autoimmune condition). 5. Currently receiving any investigational agents. 6. Known HIV-positivity 7. Uncontrolled intercurrent illness including, but not limited to ongoing infection with vital sign instability (hypotension, respiratory insufficiency), life-threatening acute tumor lysis syndrome (eg, with renal failure), symptomatic congestive heart failure, cardiac arrhythmia, intracranial or other uncontrolled bleeding, or psychiatric illness/social situations that would limit compliance with study requirements. 8. Pregnant women are excluded from this study because many of the agents used on this protocol have potential for teratogenic and/or abortifacient effects. Because there is an unknown but potential risk of adverse events in nursing infants secondary to treatment of the mother with these chemotherapy agents, breastfeeding should be discontinued if the mother is enrolled. 9. History of a previous malignancy. Exception: Individuals with a previous malignancy treated with surgery only (no chemotherapy or radiotherapy) more than 5 years prior to registration may be enrolled.

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • A Phase 1/2, Open-Label, Multi-Center, First-in-Human Study of the Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity of TPX-0005 in Patients with Advanced Solid Tumors Harboring ALK, ROS1, or NTRK1-3 Rearrangements (TRIDENT-1). - NCT03093116

    Phase 1 Dose Escalation: (1)To determine the first cycle dose-limiting toxicities (DLTs) of repotrectinib given to adult subjects with advanced solid malignancies harboring an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (2)To determine the maximum tolerated dose (MTD) of repotrectinib in adult subjects with advanced solid malignancies harboring an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (3)To determine the biologically effective dose and recommended Phase 2 dose (RP2D) of repotrectinib for adult subjects with advanced solid malignancies harboring an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. Phase 2 Study: (1)To determine the confirmed ORR as assessed by Blinded Independent Central Review (BICR) of repotrectinib in each subject population expansion cohort of advanced solid tumors that harbor an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. Phase 1 Dose Escalation: (1)To evaluate the safety and tolerability of repotrectinib at various doses in subjects with advanced solid tumors that harbor an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (2)To determine the effect of food on the pharmacokinetics (PK) of repotrectinib. (3)To determine the preliminary objective response rate by BICR (ORR) and clinical benefit rate (CBR) of repotrectinib, in subjects with advanced solid tumors that harbor an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (4)To evaluate the potential of repotrectinib to induce cytochrome P450 3A (CYP3A) using midazolam as a probe substrate. Phase 2 Study: (1)To determine the DOR, time to response (TTR), and clinical benefit rate (CBR) of repotrectinib, as assessed by BICR, in each subject population expansion cohort of advanced solid tumors that harbor an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (2)To estimate the progression-free survival (PFS) and overall survival (OS) of subjects treated with repotrectinib with advanced solid tumorsthat harbor an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (3)To evaluate the safety and tolerability of repotrectinib when administered at the RP2D in subjects with advanced solid tumors thatmharbor an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement. (4)To determine the intracranial objective response rate (IC-ORR) of repotrectinib and Central Nervous System PFS (CNS-PFS) in subjects presenting with measurable brain metastases at baseline, using RANO- BM assessment (5)To assess the population PK of repotrectinib and to explore correlations between PK, response, and/or safety findings in subjects with advanced solid tumors with an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement (6)To assess treatment-related symptoms and general health status using validated instruments of subject-reported outcomes (EORTC-QLQ-C30 and LC-13 when applicable) in subjects treated with repotrectinib

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    • Protocol Number:
      111906

    • Principal Investigator:
      Scott Moerdler M.D.

    • Phase:
      Phase I/II

    • Scope:
      National

    • Applicable Disease Sites:
      Any Site

    • Therapies Involved:
      Chemotherapy single agent systemic

    • Drugs Involved:
      Repotrectinib

      • Contacts:

      • Rutgers University Prinicipal Investigator: Scott Moerdler M.D.

    Read Inclusion & Exclusion Criteria

    PHASE 1

    Key Inclusion Criteria:

      1. Histologically or cytologically confirmed diagnosis of locally advanced, or metastatic solid tumor (including primary CNS tumors) (Stage IV, American Joint Committee on Cancer v.7) that harbors an ALK, ROS1, NTRK1, NTRK2, or NTRK3 gene rearrangement by protocol specified tests. 2. ECOG PS 0-1. 3. Age ≥18 (or age ≥ 20 of age as required by local regulation). 4. Capability to swallow capsules intact (without chewing, crushing, or opening). 5. At least 1 measurable target lesion according to RECIST version 1.1. CNS-only measurable disease as defined by RECIST version 1.1 is allowed. 6. Prior cytotoxic chemotherapy is allowed. 7. Prior immunotherapy is allowed. 8. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.03 Grade less than or equal to 1. 9. Patients with asymptomatic CNS metastases (treated or untreated) and/or asymptomatic leptomeningeal carcinomatosis are eligible to enroll if they satisfy the protocol specified criteria. 10. Baseline laboratory values fulfilling the following requirements:Absolute neutrophils count (ANC) ≥1500/mm3 (1.5 × 109/L); Platelets (PLTs) ≥100,000/mm3 (100 × 109/L); Hemoglobin ≥ 9.0 g/dL transfusions are allowed; Serum creatinine or creatinine clearance Within normal limits or > 40 mL/min; Total serum bilirubin < 1.5 × ULN; Liver transaminases (ASTs/ALTs) < 2.5 × ULN; < 5 × ULN if liver metastases are present Alkaline phosphatase (ALP); < 2.5 × ULN; < 5 × ULN if liver and/or bone metastasis are present; Serum calcium, magnesium, and potassium Normal or CTCAE grade ≤ 1 with or without supplementation 11. Life expectancy ≥ 3 months.

      PHASE 2 Key Inclusion Criteria

        1. Histologically or cytologically confirmed diagnosis of locally advanced, or metastatic solid tumor (including primary CNS tumors) that harbors a ROS1, or NTRK1-3 gene fusion. 2. Subject must have a documented ROS1 or NTRK1-3 gene fusion determined by tissue-based local testing using either: 1. a next-generation sequencing (NGS) or quantitative polymerase chain reaction (qPCR) test will be accepted to determine molecular eligibility. • Adequate tumor tissue needs to be sent to the Sponsor designated central diagnostic laboratory for retrospective confirmation by a central diagnostic laboratory test selected by the Sponsor. OR 2. a fluorescence in situ hybridization (FISH) test AND prospective confirmation of fusion status by a central diagnostic laboratory test selected by the Sponsor PRIOR to enrollment will be accepted to determine molecular eligibility.
      • Adequate tumor tissue must be sent to the Sponsor designated central diagnostic laboratory for prospective confirmation by a central diagnostic laboratory test selected by the Sponsor PRIOR to enrollment. 3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-1. 4. Age ≥12 (or age ≥ 20 as required by local regulation). 5. Willing and able to provide written institutional review board (IRB)/institutional ethics committee-approved Informed Consent or an Assent signed by a parent or legal guardian for subjects age 12 to 17. 6. At least 1 measurable target lesion according to RECIST (v1.1) prospectively confirmed by Blinded Independent Central Radiology Review (BICR), selected by Sponsor, PRIOR to enrollment. Subjects with CNS-only measurable disease ≥10 mm as defined by RECIST (v1.1) are eligible. 7. Subjects with advanced solid tumors harboring ROS1, NTRK1, NTRK2, or NTRK3 rearrangement will be assigned into 6 distinct expansion (EXP) cohorts provided all inclusion and exclusion criteria are met. i. EXP-1: ROS1 TKI-naïve ROS1+ NSCLC ii. EXP-2: 1 Prior ROS1 TKI and 1 Platinum based chemo ROS1+ NSCLC iii. EXP-3: 2 Prior ROS1 TKIs ROS1+ NSCLC (No Chemo or IO) iv. EXP-4: 1 Prior ROS1 TKI ROS1+ NSCLC (No Chemo or IO) v. EXP-5: TRK TKI-naïve NTRK+ solid tumors vi. EXP-6: TRK TKI-pretreated NTRK+ solid tumors 8. Subjects with asymptomatic CNS metastases (treated or untreated) and/or asymptomatic leptomeningeal carcinomatosis are eligible to enroll if they satisfy the protocol specified criteria. 9. Baseline laboratory values fulfilling the following requirements:Absolute neutrophils count (ANC) ≥1500/mm3 (1.5 × 109/L); Platelets (PLTs) ≥100,000/mm3 (100 × 109/L); Hemoglobin ≥ 9.0 g/dL transfusions are allowed; Serum creatinine or creatinine clearance > 40 mL/min; Total serum bilirubin < 1.5 × ULN; Liver transaminases (ASTs/ALTs) < 2.5 × ULN; < 5 × ULN if liver metastases are present Alkaline phosphatase (ALP); < 2.5 × ULN; < 5 × ULN if liver and/or bone metastasis are present; Serum calcium, magnesium, and potassium Normal or CTCAE grade ≤ 1 with or without supplementation 10. Life expectancy ≥ 3 months.

      Key Exclusion Criteria PHASE 1 and PHASE 2

        1. Concurrent participation in another therapeutic clinical trial. 2. Symptomatic brain metastases or leptomeningeal involvement. 3. History of previous cancer, except for squamous cell or basal-cell carcinoma of the skin, or any in situ carcinoma that has been completely resected, requiring therapy within the previous 2 years. 4. Major surgery within 4 weeks of start of repotrectinib treatment. Radiation therapy (except palliative to relieve bone pain) within 2 weeks of study entry. Palliative radiation (≤10 fractions) must have been completed at least 48 hours prior to study entry 5. Clinically significant cardiovascular disease (either active or within 6 months prior to enrollment): myocardial infarction, unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure (New York Heart Association Classification Class ≥ II), cerebrovascular accident or transient ischemic attack, symptomatic bradycardia, requirement for anti-arrhythmic medication. Ongoing cardiac dysrhythmias of NCI CTCAE grade ≥2 6. Any of the following cardiac criteria: Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTcF) > 470 msec obtained from 3 ECGs, using the screening clinic ECG machine-derived QTc value Any clinically important abnormalities in rhythm, conduction or morphology of resting ECG (e.g., complete left bundle branch block, third degree heart block, second degree heart block, PR interval > 250 msec) Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, hypokalemia, congenital long QT syndrome, family history of long QT syndrome, or any concomitant medication known to prolong the QT interval. 7. Known active infections (bacterial, fungal, viral including HIV positivity). 8. Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug absorption. 9. Peripheral neuropathy of CTCAE ≥grade 2. 10. History of extensive, disseminated, bilateral, or presence of CTCAE grade 3 or 4 interstitial fibrosis or interstitial lung disease including a history of pneumonitis, hypersensitivity pneumonitis, interstitial pneumonia, interstitial lung disease, obliterative bronchiolitis, and pulmonary fibrosis. Subjects with history of prior radiation pneumonitis are not excluded.

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • A Phase 1/2, Open-Label, Safety, Tolerability, Pharmacokinetics, and Anti-Tumor Activity Study of Repotrectinib in Pediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK1-3 Alterations. - NCT04094610

    Phase I: Evaluate the safety and tolerability at different dose levels of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring ALK, ROS1, or NTRK1-3 alterations to estimate the MTD or MAD and select the pediatric RP2D/schedule. Phase 2: Determine the anti-tumor activity of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring ALK, ROS1, or NTRK1-3 alterations.

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    • Protocol Number:
      111910

    • Principal Investigator:
      Scott Moerdler M.D.

    • Phase:
      Phase I/II

    • Scope:
      National

    • Applicable Disease Sites:
      Any Site

    • Therapies Involved:
      Chemotherapy single agent systemic

    • Drugs Involved:
      Repotrectinib

      • Contacts:

      • Rutgers University Prinicipal Investigator: Scott Moerdler M.D.

    Read Inclusion & Exclusion Criteria

    Key Inclusion Criteria:

      1. Documented genetic ALK, ROS1, or NTRK1-3 alteration (point mutation, fusion, amplification) as identified by local testing in a Clinical Laboratory Improvement Amendments (CLIA) laboratory in the US or equivalently accredited diagnostic lab outside the United States (US) is required. 2. Phase 1: Age <12 years; Phase 2: Age 12- 25 years 3. Prior cytotoxic chemotherapy is allowed. 4. Prior immunotherapy is allowed. 5. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.03 Grade less than or equal to 1. 6. All subjects must have measurable disease by RECIST v1.1 or Response Assessment in Neuro-Oncology Criteria (RANO) criteria at time of enrollment. 7. Subjects with a primary CNS tumor or CNS metastases must be neurologically stable on a stable or decreasing dose of steroids for at least 14 days prior to enrollment. 8. Subjects must have a Lansky (< 16 years) or Karnofsky (≥ 16 years) score of at least 50. 9. Life expectancy greater than or equal to 12 weeks. 10. Adequate hematologic, renal and hepatic function.

      Phase 2 Inclusion Criteria:

        1. Cohort Specific Inclusion Criteria:

        • Cohort 1: Subjects with NTRK fusion gene positive (NTRK+) advanced solid tumors (including primary CNS tumors), that are tropomyosin receptor kinase (TRK) TKI naïve;
        • Cohort 2: subjects with NTRK+ advanced solid tumors (including primary CNS tumors), that are TRK TKI pre-treated;
        • Cohort 3: subjects with tumors or ALCL characterized by other ALK/ROS1/NTRK alterations or NTRK fusions without centrally confirmed measurable disease or not otherwise eligible for Cohort 1 or 2. 2. Subjects in Cohorts 1 and 2 must have prospectively confirmed measurable disease by BICR prior to enrollment.

        Key Exclusion Criteria (Phase 1 and Phase 2):

          1. Subjects with neuroblastoma with only bone marrow disease evaluable by bone marrow aspiration only. 2. Major surgery within 14 days (2 weeks) of start of repotrectinib treatment. Central venous access (Broviac, Mediport, etc.) placement does not meet criteria for major surgery. 3. Known active infections (bacterial, fungal, viral including HIV positivity). 4. Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug absorption. 5. Any of the following cardiac criteria:
        • Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTc) > 480 msec obtained from three ECGs, using the screening clinic ECG machine-derived QTc value
        • Any clinically important abnormalities in rhythm, conduction, or morphology of resting ECG (e.g., complete left bundle branch block, third degree heart block, second degree heart block, PR interval > 250 msec)
        • Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, congenital long QT syndrome, family history of long QT syndrome, or any concomitant medication known to prolong the QT interval 6. Peripheral neuropathy of CTCAE ≥grade 2. 7. Subjects being treated with or anticipating the need for treatment with strong CYP3A4 inhibitors or inducers.

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • AALL1521/Incyte 18424-269: A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib with Chemotherapy in Children with De Novo High Risk CRLF-2 Rearranged and or JAK Pathway-Mutant Acute Lymphoblastic Leukemia. - NCT02723994

    1- Part 1:To evaluate initial safety and tolerability and to define the reccommended dose of ruxolitinib in combination with multi-agent chemotherapy in children and adolescents with de novo high risk Ph chromosome like cytokine receptor like factor 2 rearranged and or JAK pathway mutant B cell leukemia. 2: Part 2- to determine efficacy in combination with multi-agent chemotherapy in children and adolescents with de novo high risk Ph chromosome like cytokine receptor like factor 2 rearranged and or JAK pathway mutant B cell leukemia 3: To assess pharmacokinetics of ruxolitinib in children in combination with chemotherapy 4: To assess rates of MRD in eligible patients

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    • Protocol Number:
      111603

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase II

    • Scope:
      National

    • Applicable Disease Sites:
      Leukemia, other

    • Therapies Involved:
      Chemotherapy multiple agents systemic

    • Drugs Involved:
      METHOTREXATE VINCRISTINE THIOGUANINE CYCLOPHOSPHAMIDE MERCAPTOPURINE DOXORUBICIN DAUNORUBICIN LEUCOVORIN PEG Asparaginase (PEG) PREDNISONE DEXAMETHASONE Ruxolitinib CYTARABINE

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • Eligible for study when participant is 1 year to 21 years at the time of diagnosis
    • Eligible Ages in Australia and Canada; 2 years to 21 years
    • De novo high-risk (HR) Ph-like B-ALL for which any of following criteria are present at diagnosis:
    • Age ≥ 10 years
    • White blood cell (WBC) ≥ 50 × 10^3/μL
    • CNS3 leukemia at diagnosis
    • Systemic steroid pretreatment without presteroid WBC documentation
    • Diagnostic bone marrow or peripheral blood sample must have gene expression profiling and downstream genetic testing performed by submitting diagnostic specimens under the COG AALL08B1 or APEC14B1 biology studies, or AALL1131 or its successor study. Specimens must demonstrate a Ph-like expression profile (ie, LDA-positive) as tested by low density microarray testing at the COG ALL reference laboratory or TriCore laboratory at the University of New Mexico AND must contain 1 of the following genetic lesions: (determined at COG ALL reference laboratories, or equivalent CAP/CLIA-certified laboratories approved by the medical monitor: 1. CRLF2 rearrangement* with confirmed JAK1 or JAK2 mutation (JAK+) 2. CRLF2 rearrangement* without JAK mutation 3. Other JAK pathway alterations (eg, JAK2 fusions, EPOR fusions, SH2B3 deletions, IL7RA mutations) with or without CRLF2-R, or CRLF2-R with unknown JAK status*† as determined by a COG ALL Reference Laboratory
    • Completed a 4-drug Induction therapy regimen (modified aBFM regimen or equivalent) in Study AALL1131 or its successor study, or as per the institutional standard of care for HR B-ALL and have had end-Induction minimal residual disease (MRD) assessed
    • Male and female subjects of reproductive non childbearing potential or willing to take appropriate precautions to avoid pregnancy or fathering a child for the duration of study participation

    Exclusion Criteria:

    • Receipt of any other cytotoxic chemotherapy before Induction therapy, with exception of hydroxyurea or steroid pretreatment
    • Trisomy 21 (Down syndrome)
    • BCR-ABL1-rearranged (Ph+) ALL
    • Calculated creatinine clearance or radioisotope glomerular filtration rate < 70 mL/min/1.73 m^2
    • Alanine aminotransferase ≥ 5 × upper limit of normal (ULN) for age
    • Direct bilirubin ≥ 1.5 × ULN (may be assumed if total bilirubin is below ULN)
    • History or evidence of cirrhosis
    • Platelet count < 75 × 10^3/μL
    • Absolute neutrophil count (ANC) < 750/μL
    • Positive screen for hepatitis B or C
    • Known human immunodeficiency virus infection

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • AALL1631: International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL) Testing Imatinib in Combination with Two Different Cytotoxic Chemotherapy Backbones. - NCT03007147

    Primary Aim: To compare disease free survival (DFS) of Standard Risk (SR) pediatric Ph+ ALL treated with continuous imatinib combined with either a high-risk COG ALL chemotherapy backbone or the more intensive EsPhALL chemotherapy backbone. Secondary Aims: -To determine the feasibility of administration of imatinib after allogeneic HSCT in High Risk (HR) Ph+ ALL patients. -To determine event-free survival (EFS) of High Risk pediatric Ph+ ALL patients treated with EsPhALL chemotherapy, HSCT in first complete remission and post-HSCT imatinib. -To compare rates of Grade 3 or higher infections in SR Ph+ ALL patients between the two randomized arms. -To evaluate EFS and overall survival (OS) of all enrolled participants. To evaluate OS in SR patients. To evaluate OS in HR patients.

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    • Protocol Number:
      111804

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase III

    • Scope:
      National

    • Applicable Disease Sites:
      Any Site,Leukemia, other

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • For patients enrolled on APEC14B1 prior to enrollment on AALL1631, the required diagnostic bone marrow sample has been fulfilled
    • For patients who have not previously enrolled on APEC14B1 prior to enrollment on AALL1631, a baseline diagnostic sample (or peripheral blood sample with blasts if marrow sample unavailable) must be available to develop an MRD probe
    • In addition, laboratory reports detailing evidence of BCR-ABL1 fusion must be submitted for rapid central review within 72 hours of study enrollment
    • Newly diagnosed de novo ALL (B-ALL or T-ALL) or mixed phenotypic acute leukemia (MPAL meeting 2016 World Health Organization [WHO] definition) with definitive evidence of BCR-ABL1 fusion by karyotype, fluorescence in situ hybridization (FISH) and/or reverse transcriptase (RT)-PCR
    • Patient must have previously started induction therapy, which includes vincristine, a corticosteroid, pegaspargase, with or without anthracycline, and/or other standard cytotoxic chemotherapy
    • Patient has not received more than 14 days of multiagent induction therapy beginning with the first dose of vinCRIStine
    • Patient may have started imatinib prior to study entry but has not received more than 14 days of imatinib
    • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2
    • Direct bilirubin =< 2.0 mg/dL
    • Shortening fraction of >= 27% by echocardiogram
    • Ejection fraction of >= 50% by radionuclide angiogram or echocardiogram
    • Corrected QT interval, QTc < 480 msec
    • Note: Repeat echocardiogram is not required if echocardiogram was obtained within 21 days of study enrollment
    • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2
    • Serum creatinine within normal limits based on age/gender, as follows:
    • 1 to < 2 years: maximum serum creatinine 0.6 mg/dL (both male and female)
    • 2 to < 6 years: maximum serum creatinine 0.8 mg/dL (both male and female)
    • 6 to < 10 years: maximum serum creatinine 1 mg/dL (both male and female)
    • 10 to < 13 years: maximum serum creatinine 1.2 mg/dL (both male and female)
    • 13 to < 16 years: maximum serum creatinine 1.5 mg/dL (male), 1.4 mg/dL (female)
    • >= 16 years: maximum serum creatinine 1.7 mg/dL (male), 1.4 mg/dL (female)

    Exclusion Criteria:

    • Known history of chronic myelogenous leukemia (CML)
    • ALL developing after a previous cancer treated with cytotoxic chemotherapy
    • Active, uncontrolled infection, or active systemic illness that requires ongoing vasopressor support or mechanical ventilation
    • Down syndrome
    • Pregnancy and breast feeding
    • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential
    • Lactating females who plan to breastfeed their infants
    • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
    • Patients with congenital long QT syndrome, history of ventricular arrhythmias or heart block
    • Prior treatment with dasatinib, or any BCR-ABL1 inhibitor other than imatinib
    • All patients and/or their parents or legal guardians must sign a written informed consent
    • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Newark Beth Israel Medical Center
    • Rutgers Cancer Institute of New Jersey
  • AAML1831: A Phase 3 Randomized Trial for Patients with de novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 with GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients with FLT3 Mutations - NCT04293562

    1. To compare event-free survival (EFS) in children with de novo AML without FLT3 mutations who are randomly assigned to standard induction therapy on Arm A (DA-GO) with daunorubicin, cytarabine (DA) and gemtuzumab ozogamicin (GO) versus Arm B with CPX-351 and GO. 2. To compare overall survival (OS) and rates of end of Induction 1 (EOI1) minimal residual disease (MRD) in children with de novo AML without FLT3 mutations who are randomly assigned to standard induction therapy (Arm A) with DA-GO versus CPX-351 and GO (Arm B). 3. To estimate the EFS and rate of EOI1 MRD in FLT3 internal tandem duplication mutation positive patients (FLT3/ITD+; as defined by allelic ratio > 0.1) without favorable cytomolecular characteristics (NPM1 and/or CEBPA) receiving gilteritinib in combination with DA GO (Arm AC). 4. To estimate the EFS and rate of EOI1 MRD in patients with non-ITD FLT3 activating mutations who receive backbone therapy (DA-GO or CPX-351 and GO) with gilteritinib (Arms AD and BD). 5. To determine the feasibility of combining gilteritinib and DA-GO or CPX-351 and GO in patients with FLT3/ITD mutations (Arm AC/Arm BC/Arm AD/Arm BD). 6. To compare EOI1 MRD and EFS in patients with FLT3/ITD AML+ (AR > 0.1) without favorable cytogenetic/molecular characteristics treated with DA-GO-gilteritinib vs CPX-GO-gilteritinib (Arm AC vs Arm BC). 7. To compare the incidence of significant left ventricular systolic dysfunction (LVSD) in children with de novo AML without FLT3 mutations who are randomly assigned to standard induction therapy (Arm A) with DA-GO versus CPX-351 and GO (Arm B). 8. To compare the changes in echocardiography-derived measures of cardiac function, including left ventricular ejection fraction (EF) and global longitudinal strain (GLS), throughout AML therapy in patients with low and high risk AML without FLT3 mutations receiving Arm A vs Arm B. 9. Determine if early changes in sensitive echocardiographic measures of cardiac function (i.e., post-Induction 1 decline in GLS) and elevations in circulating cardiac biomarkers (i.e., cardiac troponin T and N-terminal pro b-type natriuretic peptide) are associated with subsequent declines in left ventricular ejection fraction in patients with non-FLT3 mutant AML receiving therapy on Arms A or B. 10. To compare longitudinal acute changes in neuropsychological functioning and neurocognitive late effects between those with CNS disease and those without CNS disease and between those treated with HSCT and those treated with chemotherapy only for patients on Arms A and B. 11. To compare cardiotoxicity measures (EF, GLS, and cardiac biomarkers) in patients receiving standard induction with dexrazoxane vs. CPX-351 in the context of gilteritinib therapy and explore whether the differential cardiotoxicity across arms varies from that observed in non-FLT3 mutant AML without gilteritinib exposure. 12. To estimate the EFS and rate of EOI1 MRD in patients with high allelic ratio (HAR) FLT3/ITD+ patients, as historically defined by an AR > 0.4, receiving gilteritinib in combination with DA-GO (Arm AC with AR > 0.4). 13. To estimate the EFS and rate of EOI1 MRD in FLT3/ITD+ patients (as defined by allelic ratio > 0.1) with NPM1 and/or bZIP CEBPA mutations receiving gilteritinib in combination with DA-GO (Arm AC). 14. Compare the changes in high sensitivity troponin and natriuretic peptide elevations throughout AML therapy, as measured at the end of each chemotherapy course, in patients with low and high risk AML without FLT3 mutations receiving Arm A vs Arm B

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    • Protocol Number:
      112008

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase III

    • Scope:
      National

    • Applicable Disease Sites:
      Leukemia, not otherwise specified

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831. Submission of diagnostic specimens must be done according to the Manual of Procedures
    • Patients must be less than 22 years of age at the time of study enrollment
    • Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease
    • Patient must have 1 of the following:
    • >= 20% bone marrow blasts (obtained within 14 days prior to enrollment)
    • In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
    • < 20% bone marrow blasts with one or more of the genetic abnormalities associated with childhood/young adult AML as provided in the protocol (sample obtained within 14 days prior to enrollment)
    • A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell [WBC] count >= 10,000/uL with >= 10% blasts or a WBC count of >= 5,000/uL with >= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (performed within 7 days prior to enrollment)
    • ARM C: Patient must be >= 2 years of age at the time of Late Callback
    • ARM C: Patient must have FLT3/ITD allelic ratio > 0.1 as reported by Molecular Oncology
    • ARM C: Patient does not have any congenital long QT syndrome or congenital heart block
    • ARM C: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
    • ARM C: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
    • ARM C: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
    • ARM D: Patient must be >= 2 years of age at the time of Late Callback
    • ARM D: Patient must have one of the clinically relevant non-ITD FLT3 activating mutations as reported by Foundation Medicine
    • ARM D: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
    • ARM D: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
    • ARM D: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
    • NEUROPSYCHOLOGICAL TESTING: Patient must be enrolled on Arm A or Arm B. Patients who transfer to Arm C or Arm D are not eligible
    • NEUROPSYCHOLOGICAL TESTING: Patient must be 5 years or older at the time of enrollment
    • NEUROPSYCHOLOGICAL TESTING: English-, French- or Spanish-speaking
    • NEUROPSYCHOLOGICAL TESTING: No known history of neurodevelopmental disorder prior to diagnosis of AML (e.g., Down syndrome, fragile X, William syndrome, mental retardation)
    • NEUROPSYCHOLOGICAL TESTING: No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
    • All patients and/or their parents or legal guardians must sign a written informed consent
    • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.

    Exclusion Criteria:

    • Fanconi anemia
    • Shwachman Diamond syndrome
    • Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
    • Telomere disorders
    • Germline predispositions known, or suspected by the treating physician to increase risk of toxicity with AML therapy
    • Any concurrent malignancy
    • Juvenile myelomonocytic leukemia (JMML)
    • Philadelphia chromosome positive AML
    • Mixed phenotype acute leukemia
    • Acute promyelocytic leukemia
    • Acute myeloid leukemia arising from myelodysplasia
    • Therapy-related myeloid neoplasms
    • Patients with persistent cardiac dysfunction prior to enrollment, defined as ejection fraction (EF) < 50% (preferred method Biplane Simpson's EF) or if EF unavailable, shortening fraction (SF) < 24%. *Note: if clinically safe and feasible, repeat echocardiogram is strongly advised in order to confirm cardiac dysfunction following clinical stabilization, particularly if occurring in the setting of sepsis or other transient physiologic stressor. If the repeat echocardiogram demonstrates an EF >= 50%, the patient is eligible to enroll and may receive an anthracycline-containing Induction regimen
    • Administration of prior anti-cancer therapy except as outlined below:
    • Hydroxyurea
    • All-trans retinoic acid (ATRA)
    • Corticosteroids (any route)
    • Intrathecal therapy given at diagnosis
    • In particular, strong inducers of CYP3A4 and/or P-glycoprotein (P-gp) should be avoided from the time of enrollment until it is determined whether the patient will receive gilteritinib. Patients receiving gilteritinib will be required to avoid strong CYP3A4 inducers and/or strong P-gp inducers for the duration of the study treatment
    • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
    • Lactating females who plan to breastfeed their infants
    • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
    • ARM D: Patient does not have any congenital long QT syndrome or congenital heart block

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Newark Beth Israel Medical Center
    • Rutgers Cancer Institute of New Jersey
  • AAML18P1: Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP). - NCT03817398

    1. To determinethe 2-year treatment free remission (TFR) rate of children,adolescents,and young adults with CML-CP following discontinuation TKI. 2. To estimate the re-induction rate and maintenance of major molecular remission (MMR/MR3) at 1 year after restarting TKI for children, adolescents, and young adults. 3.To describe clinical factors and laboratory correlates affecting the persistence of MMR and re-initiation of treatment after stopping TKI (e.g. patient demographics, duration and level of prior molecular remission, duration and type of TKI, clinical presentation at diagnosis and immune studies) 4. To describe change in height standard deviation score over time in patients who are able to discontinue their TKI. 5. To describe the long-term health outcomes including but not limited to gonadal function, endocrine function, and bone metabolism in patients who are able to discontinue TKI as well as those that need to restart TKIs. 6. To describe differences in patient-reported health status after stopping TKIs, including those who need to resume TKI after stopping. 7. To describe the incidence and characteristics of TKI withdrawal syndrome in children. 8.To evaluate changes in neurocognitive outcomes of patients enrolled on this studyusing a patient-completed, performance-based, computerized measure of neuropsychological functioning and a parent-report/self-report questionnaire.

    View All Details
    • Protocol Number:
      111905

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase II

    • Scope:
      National

    • Applicable Disease Sites:
      Leukemia, other

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • Patient must have been diagnosed with CML-CP at < 18 years of age.
    • Patient must have histologic verification of CML-CP at original diagnosis
    • Patient must be in molecular remission (MR) with a BCR-ABL1 level of =< 0.01% BCR-ABL1 as measured using the International Scale (IS) by RQ-PCR for >= 2 consecutive years at the time of enrollment
    • Please note: The lab evaluating disease status and molecular response for this study must be College of American Pathology (CAP) and/or Clinical Laboratory Improvement Amendments (CLIA) certified (United States [US] only), sites in other countries must be certified by their accredited authorities. All labs must use the International Scale guidelines with a sensitivity of detection assay =< 0.01% BCR-ABL1 and be able to report results in =< 2 weeks
    • Patient must have received any TKI for a minimum of 3 consecutive years at time of enrollment
    • Patient agrees to discontinue TKI therapy
    • REGULATORY REQUIREMENTS
    • All patients and/or their parents or legal guardians must sign a written informed consent
    • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
    • ELIGIBILITY FOR PATIENT-REPORTED OUTCOMES (PROs):
    • Age >= 8 years at the time of enrollment
    • Ability to understand English or Spanish
    • Cognitive ability to complete instruments according to the primary team
    • ELIGIBILITY FOR AAML18P1 NEUROCOGNITIVE STUDY:
    • Patient must be 5 years or older at the time of enrollment
    • English-, French- or Spanish-speaking
    • No known history of neurodevelopmental disorder prior to diagnosis of CML (e.g., Down syndrome, Fragile X, William syndrome, mental retardation)
    • No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli

    Exclusion Criteria:

    • Known T3151 mutation
    • Additional clonal chromosomal abnormalities in Philadelphia chromosome (Ph) positive (+) cells at any time prior to enrollment that include "major route" abnormalities (second Ph, trisomy 8, isochromosome 17q, trisomy 19), complex karyotype or abnormalities of 3q26.2
    • History of accelerated phase or blast crisis CML
    • Female patients who are pregnant
    • Lactating females are not eligible unless they have agreed not to breastfeed their infants
    • Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • ACNS1831: A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma. - NCT03871257

    1: To determine whether the efficacy of treatment with selumetinib as measured by event-free survival (EFS) is non-inferior to treatment with carboplatin/vincristine (CV) in previously untreated Neurofibromatosis type 1 (NF1)-associated low- grade glioma (LGG). 2: To determine whether visual acuity (VA) using Teller Acuity Cards (TAC), in patients with NF1-associated LGG within the optic pathway, is better in those treated with selumetinib. 3: To estimate tumor response rates and overall survival (OS) in each treatment regimen in previously untreated NF1-associated LGG. 4: To evaluate VA outcomes utilizing HOTV letter acuity testing in previously untreated NF1-associated LGG within the optic pathway in patients who are old enough to perform visual acuity testing utilizing HOTV (a recognition acuity measure). 5: To describe the improvement in motor function as measured by the Vineland scale in patients with previously untreated NF1-associated LGG that have documented motor deficits at enrollment. 6: To prospectively evaluate and compare the quality of life among patients treated with selumetinib or CV. 7: To prospectively evaluate and compare the cognitive, social, emotional, and behavioral functioning of patients with NF1-associated LGG treated with either selumetinib or CV. 8: To evaluate optical coherence tomography (OCT) measures of retinal axon and ganglion cell thickness as a marker of treatment response in previously untreated NF1-associated LGG within the optic pathway. 9: To compare novel, semi-automated volumetric MRI measures to traditional measurements of treatment response (bi-dimensional MRI measurements) in NF1- associated optic pathway tumors. 10: To obtain paired blood and tumor tissue to be banked for future NF1-LGG biology studies involving comprehensive molecular analysis, including but not limited to whole exome and RNA sequencing.

    View All Details
    • Protocol Number:
      111914

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase III

    • Scope:
      National

    • Applicable Disease Sites:
      Brain and Nervous System

    • Therapies Involved:
      Chemotherapy multiple agents systemic Chemotherapy single agent systemic

    • Drugs Involved:
      CARBOPLATIN Selumetinib VINCRISTINE

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
    • Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing
    • Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery
    • For patients with optic pathway gliomas (OPGs):
    • Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms (including visual dysfunction, as defined below) or other exam findings associated with the tumor
    • Previously-diagnosed patients with OPG are eligible if they have new or worsening neurologic symptoms (including visual dysfunction, as defined below) or have tumor growth
    • For both newly-diagnosed and previously-diagnosed OPG, the patient may be eligible, irrespective of whether there has been tumor growth or other neurological symptoms or worsening, if they meet at least one of the following visual criteria:
    • Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR
    • Significant visual dysfunction (defined as VA worse than normal for age by 0.6 logMAR [20/80, 6/24, or 2.5/10] or more in one or both eyes)
    • For patients with LGG in other locations (i.e., not OPGs):
    • Newly-diagnosed patients with LGG are eligible if there are neurologic symptoms or other exam findings associated with the tumor
    • NOTE: Newly-diagnosed patients with LGG without associated neurologic symptoms or exam findings are not eligible
    • Previously-diagnosed patients with LGG are eligible if they have new or worsening neurologic symptoms or have tumor growth
    • Although not required, if a biopsy/tumor resection is performed, eligible histologies will include all tumors considered LGG or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
    • Patients must have two-dimensional measurable tumor >= 1 cm^2
    • Patients with metastatic disease or multiple independent primary LGGs are allowed on study
    • Creatinine clearance or radioisotope glomerular filtration Rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender within 7 days prior to enrollment as follows:
    • Age; maximum serum creatinine (mg/dL)
    • 2 to < 6 years; 0.8 (male) and 0.8 (female)
    • 6 to < 10 years; 1 (male) and 1 (female)
    • 10 to < 13 years; 1.2 (male) and 1.2 (female)
    • 13 to < 16 years; 1.5 (male) and 1.4 (female)
    • >= 16 years; 1.7 (male) and 1.4 (female)
    • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age within 7 days prior to enrollment (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
    • Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x upper limit of normal (ULN) = 135 U/L within 7 days prior to enrollment. For the purpose of this study, the ULN for SGPT is 45 U/L
    • Albumin >= 2 g/dL within 7 days prior to enrollment
    • Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram within 7 days prior to enrollment
    • Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) within 7 days prior to enrollment
    • Absolute neutrophil count >= 1,000/uL (unsupported) within 7 days prior to enrollment
    • Platelets >= 100,000/uL (unsupported) within 7 days prior to enrollment
    • Hemoglobin >= 8 g/dL (may be supported) within 7 days prior to enrollment
    • Patients with a known seizure disorder should be stable and should have not experienced a significant increase in seizure frequency within 2 weeks prior to enrollment
    • Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment. Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of antihypertensive medications).
    • Note: Adequate blood pressure can be achieved using medication for the treatment of hypertension
    • All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
    • For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment
    • For patients who undergo a surgery on the target tumor (not required), a pre- and post-operative* MRI of the brain (with orbital cuts for optic pathway tumors) or spine (depending on the site(s) of primary disease) with and without contrast must also be performed within 4 weeks prior to enrollment
    • The post-operative MRIs should be performed ideally within 48 hours after surgery if possible
    • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
    • Patients must have the ability to swallow whole capsules
    • Patients must have receptive and expressive language skills in English or Spanish to complete the quality of life (QOL) and neurocognitive assessments
    • All patients and/or their parents or legal guardians must sign a written informed consent.
    • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.

    Exclusion Criteria:

    • Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
    • Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
    • Patients may not be receiving any other investigational agents
    • Patients with any serious medical or psychiatric illness/ condition, including substance use disorders likely in the judgement of the investigator to interfere or limit compliance with study requirements/treatment are not eligible
    • Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
    • Female patients who are pregnant are not eligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
    • Lactating females who plan to breastfeed their infants are not eligible
    • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible
    • Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo
    • Cardiac conditions:
    • Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
    • Symptomatic heart failure
    • New York Heart Association (NYHA) class II-IV prior or current cardiomyopathy
    • Severe valvular heart disease
    • History of atrial fibrillation
    • Ophthalmologic conditions:
    • Current or past history of central serous retinopathy
    • Current or past history of retinal vein occlusion or retinal detachment
    • Patients with uncontrolled glaucoma
    • If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
    • Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor, or strabismus) or longstanding orbito-temporal plexiform neurofibroma (PN, such as visual loss, strabismus) will NOT be considered a significant abnormality for the purposes of the study
    • Treatments and/or medications patient is receiving that would make her/him ineligible, such as:
    • Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E
    • Surgery within 2 weeks prior to enrollment, with the exception of surgical placement for vascular access or cerebrospinal fluid (CSF) diverting procedures such as endoscopic third ventriculostomy (ETV) and ventriculo-peritoneal (VP) shunt.
    • Note: Patients must have healed from any prior surgery prior to enrollment
    • Patients who have an uncontrolled infection are not eligible

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • AGAVE-201, A Phase 2, Open-label, Randomized, Multicenter Study to Evaluate the Efficacy, Safety and Tolerability of Axatilimab at 3 Different Doses in Patients with Recurrent or Refractory Active Chronic Graft Versus Host Disease who have Received at least 2 Lines of Systemic Therapy. - NCT04710576

    Primary Objective: To evaluate the overall response rate (ORR) of axatilimab at 0.3 mg/kg IV Q2W, 1 mg/kg IV Q2W, and 3 mg/kg IV Q4W in patients with cGVHD after failure of least 2 prior lines of therapy. Secondary - Efficacy Objectives: To evaluate key secondary measures of clinical benefit- Proportion of patients with a >5-point improvement in modified Lee Symptom Scale score (mLSS). Secondary - Measures of clinical benefit: 1. ORR on study as defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD. 2. Duration of response (DOR) defined as the time from best response of PR or CR until documented progression of cGVHD, start of new therapy, or death for any reason (Definition 1). 3. DOR defined as the time from initial response of PR or CR until documented progression of cGVHD, start of new therapy, or death for any reason (Definition 2). 4. Sustained response rate (SRR). 5. Organ-specific response rate based on 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD. 6. Joints and fascia response rate based on refined NIH response algorithm for cGVHD. 7. Percent reduction in average daily dose (or equivalent) of corticosteroids 8. Proportion of patients who discontinue corticosteroid use after study entry. 9. Percent reduction in average daily dose (or equivalent) of calcineurin inhibitors. 10. Proportion of patients who discontinue calcineurin inhibitors use after study entry. Secondary - Safety: 1. To evaluate the safety and tolerability of axatilimab in patients with cGVHD. 2. Bone morphology: Change from baseline in bone turnover markers. Change from baseline in bone density. Secondary - PK/PD: 1. To assess the plasma population PK (pop PK) profile of axatilimab in patients with cGVHD. To assess pharmacodynamic profile of axatilimab. 2. To determine or assess the changes in monocyte level with response. 3. To determine or assess the baseline in monocyte level with response. Secondary - Immunogenicity: Presence of anti-drug antibody (ADA).

    View All Details
    • Protocol Number:
      022103

    • Principal Investigator:
      Roger Strair M.D., Ph.D.

    • Phase:
      Phase II

    • Scope:
      National

    • Applicable Disease Sites:
      Leukemia, other,Leukemia, not otherwise specified

    • Therapies Involved:
      Chemotherapy single agent systemic

    • Drugs Involved:
      Axatilimab

      • Contacts:

      • Rutgers University Prinicipal Investigator: Roger Strair M.D., Ph.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

      1. Participants must be 2 years of age or older, at the time of signing the informed consent. 2. Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression. Active cGVHD is defined as the presence of signs and symptoms of cGVHD per 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD. 3. Participants with refractory or recurrent active cGVHD despite at least 2 lines of systemic therapy.
    • Refractory disease defined as meeting any of the following criteria:
    • The development of 1 or more new sites of disease while being treated for cGVHD.
    • Progression of existing sites of disease despite at least 1 month of standard or investigation therapy for cGVHD.
    • Participants who have not achieved a response within 3 months on their prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.
    • Recurrent cGVHD is active, symptomatic disease (after an initial response to prior therapy) as defined, based on the NIH 2014 consensus criteria, by organ-specific or global assessment or for which the physician believes that a new line of systemic therapy is required. 4. Participants may have persistent, active acute and cGVHD manifestations (overlap syndrome), as defined by 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD. 5. Karnofsky Performance Scale of ≥60 (if aged 16 years or older); Lansky Performance Score of ≥60 (if aged <16 years) 6. Adequate organ and bone marrow functions evaluated during the 14 days prior to randomization. 7. Creatinine clearance (CrCl) ≥30 milliliter/minute/1.73 square meter based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants. 8. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. 9. Concomitant use a of systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking corticosteroids at study randomization, they must be on a stable dose of corticosteroids for at least 2 weeks prior to Cycle 1 Day 1. 10. Concomitant use of CNI or sirolimus is allowed but not required. 11. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. A parent/guardian should provide consent for pediatric participants unable to provide consent themselves; in addition, where applicable pediatric participants should sign their own assent form.

    Exclusion Criteria:

      Participants are excluded from the study if any of the following criteria apply: 1. Has acute GVHD without manifestations of cGVHD. 2. Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening. 3. History of acute or chronic pancreatitis. 4. History of myositis. 5. History or other evidence of severe illness, uncontrolled infection or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study. 6. Participants with acquired immune deficiency syndrome (AIDS). 7. Hepatitis B (defined as hepatitis B virus [HBV] surface antigen positive and HBV core antibody positive, with positive HBV deoxyribonucleic acid [DNA], or HBV positive core antibody alone with positive HBV DNA. Hepatitis C (defined as positive hepatitis C [HCV] antibody with positive HCV ribonucleic acid [RNA]). 8. Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years of randomization, unless previously treated with curative intent and approved by Sponsor's Medical Monitor (for example, completely resected basal cell or squamous cell carcinoma of the skin, resected in situ cervical malignancy, resected breast ductal carcinoma in situ, or low-risk prostate cancer after curative resection). 9. Female participant who is pregnant or breastfeeding. 10. Previous exposure to CSF1-R targeted therapies. 11. Taking agents for treatment of cGVHD other than corticosteroids and either a CNI or sirolimus is prohibited. 12. For approved or commonly used agents, other than corticosteroids, CNI and sirolimus, a washout of 2 weeks or 5 half-lives, whichever is shorter, is required at study enrollment. 13. Receiving another investigational treatment within 28 days of randomization. 14. Participants should not be participating in any other interventional study. Pediatric participants are encouraged to also participate in the ongoing developmental studies of the Pediatric cGVHD Symptom Scale (PCSS).

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Rutgers Cancer Institute of New Jersey
  • AGCT1531: A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors. - NCT03067181

    The aim of this study for low and standard risk germ cell tumor (GCT) patients is to minimize toxicity by reducing therapy while maintaining current survival rates. The trial will eliminate chemotherapy for low risk patients who are likely cured with surgery and will observe the salvage rates among those who recur.

    View All Details
    • Protocol Number:
      111702

    • Principal Investigator:
      Archana Sharma DO

    • Phase:
      Phase III

    • Scope:
      National

    • Applicable Disease Sites:
      Any Site,Prostate,Ovary

    • Therapies Involved:
      Chemotherapy multiple agents systemic

    • Drugs Involved:
      BLEOMYCIN CARBOPLATIN CISPLATIN ETOPOSIDE

      • Contacts:

      • Rutgers University Prinicipal Investigator: Archana Sharma DO

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • There is no age limit for the low risk stratum (stage I ovarian immature teratoma and stage I non-seminoma or seminoma malignant GCT [all sites])
    • Standard risk 1: Patient must be < 11 years of age at enrollment
    • Standard risk 2: Patients must be >= 11 and < 25 years of age at enrollment
    • Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I germ cell tumor; for the standard risk arms, patients must be newly diagnosed with metastatic germ cell tumor (stage II or higher); histologic confirmation of a primary extracranial germ cell tumor in any of the categories outlined below is required of all patients at enrollment except for those who were initially diagnosed with stage I non-seminoma malignant GCT and later recur during observation post surgery off study; for these patients, if elevated tumor markers rise to > 5 x upper limit of normal (ULN) on at least 2 measurements taken at least 1 week apart, a diagnostic biopsy is not required for enrollment
    • Low risk stage I immature teratoma (IT); site: ovarian; stage: Children's Oncology Group (COG) stage I, Federation of Gynecology and Obstetrics (FIGO) stage IA and IB; grade: 2 or 3; histology: pure immature teratoma (may contain microscopic foci of yolk sac tumor), mixed immature and mature teratoma, (no pathological evidence of MGCT); tumor markers: alpha-FP =< 1,000 ng/mL, beta-HCG institutional normal; all ages
    • Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage: COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC) testicular stage IA, IB and IS; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
    • Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular stage IA IB, and IS; histology: must contain at least one of the following: may contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma, or choriocarcinoma; all ages
    • Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage II-IV, FIGO stage IC, FIGO stage II-IV (International Germ Cell Consensus Classification [IGCCC] criteria DO NOT apply); histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) < 11
    • Standard risk 2 (SR2)
    • Site: ovarian; stage: COG stage II and III, FIGO stage IC, II and III; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) >= 11 and < 25
    • Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; tumor markers: must be IGCCC good risk; post op: alpha-FP < 1,000 ng/mL, beta-HCG < 5,000 IU/mL and lactate dehydrogenase (LDH) < 3.0 x normal; age (years) >= 11 and < 25
    • Site: extragonadal; stage: COG stage II; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) >= 11 and < 25
    • Notes:
    • IGCCC criteria only apply to SR2 patients with a testicular primary tumor
    • Use post-op tumor marker levels to determine IGCCC risk group
    • Stage 1 seminoma patients are not eligible for the standard risk arms of the study
    • For the low risk stage I non-seminoma MGCT and the standard risk arms, components of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac tumor is the only malignant component present, then it must be deemed by the pathologist to be greater than a "microscopic component" of yolk sac tumor
    • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
    • Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1 and SR2 patients)
    • Adequate renal function defined as:
    • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 OR
    • A serum creatinine based on age/gender as follows: (mg/dL)
    • 1 month to < 6 months male: 0.4 female: 0.4
    • 6 months to < 1 year male: 0.5 female: 0.5
    • 1 to < 2 years male: 0.6 female: 0.6
    • 2 to < 6 years male: 0.8 female: 0.8
    • 6 to < 10 years male: 1 female: 1
    • 10 to < 13 years male: 1.2 female: 1.2
    • 13 to < 16 years: male: 1.5 female: 1.4
    • >= 16 years male: 1.7 female: 1.4
    • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
    • Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x upper limit of normal (ULN) for age (for the purpose of this study, the ULN for SGPT is 45 U/L)
    • Peripheral absolute neutrophil count (ANC) >= 1,000/mm^3 AND
    • Platelet count >= 100,000/mm^3
    • Patients enrolling on the standard risk arms must be medically fit to receive protocol treatment and with no contraindications to protocol treatment
    • Eligibility criteria to participate in the pilot study of the AYA-Hears instrument (patient reported outcomes [PROs] of ototoxicity) Note: participants in group 1 will not receive AGCT1531 protocol-directed therapy; all other AYA-HEARS patients must be enrolled on the AGCT1531 SR2 arm in order to participate
    • >= 11 and < 25 years old at enrollment
    • Able to fluently speak and read English
    • Has received prior cisplatin- or carboplatin-based chemotherapy regimen for malignancy including diagnoses other than germ cell tumor
    • Followed for cancer or survivorship care at one of the following institutions:
    • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
    • Dana Farber/Harvard Cancer Center
    • Hospital for Sick Children
    • Children's Hospital of Eastern Ontario
    • Oregon Health and Science University
    • Seattle Children's Hospital
    • Yale University

    Exclusion Criteria:

    • Patients with any diagnoses not listed including:
    • Stage I testicular cancer patients who have undergone primary RPLND (retroperitoneal lymph node dissection)
    • Pure dysgerminoma
    • Pure mature teratoma
    • Pure immature teratoma COG stage I, grade I
    • Pure immature teratoma COG stage I, grade 2,3 with alpha-fetoprotein (AFP) >= 1000 ng/mL
    • Pure immature teratoma COG stage II - IV or FIGO stage IC to IV
    • "Poor risk" GCT (age >= 11 years old and COG stage IV ovarian, COG stage III or IV EG, or IGCCC intermediate or poor risk testicular), or
    • Primary central nervous system (CNS) germ cell tumor
    • Germ cell tumor with somatic malignant transformation
    • Spermatocytic seminoma
    • Patients must have had no prior systemic therapy for the current cancer diagnosis
    • Patients must have had no prior radiation therapy with the exception of CNS irradiation of brain metastases; (this exception only applies to SR1 patients; any patients over age 11 with distant metastases to brain [stage IV disease] would be considered poor risk and therefore not eligible for this trial)
    • Patients with significant, pre-existing co-morbid respiratory disease that contraindicate the use of bleomycin are ineligible for the standard risk arms of the trial
    • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
    • Lactating females who plan to breastfeed their infants; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
    • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Newark Beth Israel Medical Center
    • Rutgers Cancer Institute of New Jersey
  • AHEP1531: Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT) (Childrens Oncology Group). - NCT03533582

    1.1.1 To reduce therapy associated toxicity for patients with non-metastatic hepatoblastoma (HB) and hepatocellular carcinoma (HCC) without adversely affecting long term outcomes. 1.1.1.1 GroupA:To determinetheevent-freesurvival(EFS)inpatientswithHB whose tumor is completely resected at diagnosis and either receive no adjuvant chemotherapy (completely resected well differentiated fetal [WDF] histology HB) or 2 cycles of standard dose cisplatin monotherapy (completely resected non-well differentiated fetal histology HB 100 mg/m2/cycle given 3 weeks apart). 1.1.1.2 Group B: To demonstrate that 4 to 6 cycles of interval compressed lower dose cisplatin monotherapy (80 mg/m2/cycle; 320-480 mg/m2 total) is adequate for Low Risk HB. a. In patients who are resected after 2 cycles of cisplatin monotherapy, to compare EFS following a randomized comparison of 2 versus 4 post-operative cycles of cisplatin monotherapy. b. In patients whose tumors are deemed unresectable after 2 cycles of cisplatin monotherapy, to determine the proportion of tumors rendered completely resectable by an additional 2 or 4 cycles of chemotherapy. 1.1.1.3 Group C: To compare in a randomized fashion, EFS in patients with Intermediate Risk HB treated with 6 cycles of cisplatin/5- fluorouracil/vincristine/doxorubicin (C5VD) chemotherapy versus 6 cycles of interval compressed cisplatin monotherapy (100 mg/m2/dose). 1.1.1.4 Group E: To determine the EFS in patients with HCC whose tumor is completely resected at diagnosis who receive no adjuvant chemotherapy (completely resected HCC arising in the context of underlying liver disease) or 4 cycles of cisplatin/doxorubicin (PLADO) (completely resected de novo HCC). 1.1.2 To improve the EFS of patients with High Risk HB (Group D) by treating them with interval compressed cisplatin and doxorubicin based induction regimen followed by response-adapted consolidation therapy. 1.1.2.1 Group D1 In patients whose metastatic disease resolves with the administration of SIOPEL 4 Induction therapy, to determine if the promising pilot results observed in SIOPEL 4 can be validated in a large international study. 1.1.2.2 Group D Arm CE & Arm VI In patients whose metastatic disease does not resolve with the administration of SIOPEL 4 Induction therapy, to determine in a randomized comparison which post induction treatment (irinotecan and vincristine alternating with carboplatin and doxorubicin or carboplatin and etoposide alternating with carboplatin and doxorubicin) results in superior outcomes. 1.1.3 In patients with unresectable/metastatic HCC at diagnosis (Group F), to determine whether the addition of gemcitabine and oxaliplatin (GEMOX + sorafenib) to a cisplatin, doxorubicin and sorafenib backbone improves chemotherapy response, resectability and survival.

    View All Details
    • Protocol Number:
      111806

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase II/III

    • Scope:
      National

    • Applicable Disease Sites:
      Liver

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • Patients in Group F must have a body surface area (BSA) >= 0.6 m^2
    • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
    • Patients must be newly diagnosed with histologically-proven primary pediatric hepatic malignancies including hepatoblastoma or hepatocellular carcinoma, except as noted below; patients with a diagnosis of hepatocellular neoplasm, not otherwise specified, should be classified and treated per hepatoblastoma treatment arms; note that rapid central pathology review is required in some cases; please note: all patients with histology as assessed by the institutional pathologist consistent with pure small cell undifferentiated (SCU) HB will be required to have testing for INI1/SMARCB1 by immunohistochemistry (IHC) according to the practices at the institution
    • Patients with histology consistent with pure SCU must have positive INI1/SMARCB1 staining
    • For all Group A patients, WDF status as determined by rapid review will be used to further stratify patients to Group A1 or A2
    • For Groups B, C and D, rapid review is required if patients are either >= 8 years of age or have an alphafetoprotein (AFP) =< 100 at diagnosis
    • For all Groups E and F patients, rapid central pathology review is required
    • In emergency situations when a patient meets all other eligibility criteria and has had baseline required observations, but is too ill to undergo a biopsy safely, the patient may be enrolled without a biopsy
    • Clinical situations in which emergent treatment may be indicated include, but are not limited to, the following circumstances:
    • Anatomic or mechanical compromise of critical organ function by tumor (e.g., respiratory distress/failure, abdominal compartment syndrome, urinary obstruction, etc.)
    • Uncorrectable coagulopathy
    • For a patient to maintain eligibility for AHEP1531 when emergent treatment is given, the following must occur:
    • The patient must have a clinical diagnosis of hepatoblastoma, including an elevated alphafetoprotein (AFP), and must meet all AHEP1531 eligibility criteria at the time of emergent treatment
    • Patient must be enrolled on AHEP1531 prior to initiating protocol therapy; a patient will be ineligible if any chemotherapy is administered prior to AHEP1531 enrollment
    • Note: If the patient receives AHEP1531 chemotherapy emergently PRIOR to undergoing a diagnostic biopsy, pathologic review of material obtained in the future during either biopsy or surgical resection must either confirm the diagnosis of hepatoblastoma or not reveal another pathological diagnosis to be included in the analysis of the study aims
    • Patients may have had surgical resection of the hepatic malignancy prior to enrollment; all other anti-cancer therapy for the current liver lesion is prohibited
    • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 60 mL/min/1.73 m^2 or
    • A serum creatinine based on age/gender as follows:
    • Age: maximum serum creatinine (mg/dL)
    • 1 month to < 6 months: 0.4 (male and female)
    • 6 months to < 1 year: 0.5 (male and female)
    • 1 to < 2 years: 06 (male and female)
    • 2 to < 6 years: 0.8 (male and female)
    • 6 to < 10 years: 1 (male and female)
    • 10 to < 13 years: 1.2 (male and female)
    • 13 to < 16 years: 1.5 (male), 1.4 (female)
    • >= 16 years: 1.7 (male), 1.4 (female)
    • Total bilirubin =< 5 x upper limit of normal (ULN) for age
    • Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) < 10 x upper limit of normal (ULN) for age
    • Shortening fraction of >= 28% by echocardiogram (for patients on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior to study enrollment) or
    • Ejection fraction of >= 47% by echocardiogram or radionuclide angiogram (for patients on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior to study enrollment)
    • Group F patients only: QT/corrected QT (QTc) interval =< 450 milliseconds for males and =< 470 milliseconds for females (assessed within 8 weeks prior to study enrollment)
    • Normal pulmonary function tests (including diffusion capacity of the lung for carbon monoxide [DLCO]) if there is clinical indication for determination (e.g. dyspnea at rest, known requirement for supplemental oxygen) (for patients receiving chemotherapy [Groups A, B, C, D, E2, F]); for patients who do not have respiratory symptoms or requirement for supplemental oxygen, pulmonary function tests (PFTs) are NOT required
    • All patients and/or their parents or legal guardians must sign a written informed consent
    • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

    Exclusion Criteria:

    • Prior chemotherapy or tumor directed therapy (i.e. radiation therapy, biologic agents, local therapy (embolization, radiofrequency ablation, and laser); therefore, patients with a pre-disposition syndrome who have a prior malignancy are not eligible
    • Patients who are currently receiving another investigational drug
    • Patients who are currently receiving other anticancer agents
    • Patients with uncontrolled infection
    • Patients who previously received a solid organ transplant, other than those who previously received an orthotopic liver transplantation (OLT) as primary treatment of their hepatocellular carcinoma
    • Patients with hypersensitivity to any drugs on their expected treatment arm
    • Group C: Patients who have known deficiency of dihydropyrimidine dehydrogenase (DPD)
    • Group D:
    • Patients with chronic inflammatory bowel disease and/or bowel obstruction
    • Patients with concomitant use of St. John's wort, which cannot be stopped prior to the start of trial treatment
    • Group F:
    • Patients with peripheral sensitive neuropathy with functional impairment
    • Patients with a personal or family history of congenital long QT syndrome
    • These criteria apply ONLY to patients who may receive chemotherapy (all groups other than Group E1):
    • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential
    • Lactating females who plan to breastfeed their infants
    • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
    • Note for Group F: patients of childbearing potential should use effective birth control during treatment with sorafenib and for at least 2 weeks after stopping treatment

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Newark Beth Israel Medical Center
    • Rutgers Cancer Institute of New Jersey
  • ANBL1531: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379) - NCT03126916

    Primary Aims 1.1.1 To determine in the context of a randomized trial whether the EFS of patients with newly diagnosed high-risk NBL is improved with the addition of 131I-MIBG during Induction, prior to tandem autologous stem cell transplantation (ASCT). 1.1.2 To determine whether the addition of crizotinib to intensive multimodality therapy for patients with high-risk NBL whose tumors harbor activating point mutations in or amplification of the ALK gene results in superior EFS compared to a contemporaneously treated cohort of patients whose tumors lack these ALK aberrations. 1.2 Secondary Aims 1.2.1 To describe the toxicities associated with treatment for high-risk NBL with and without the addition of 131I-MIBG or crizotinib. 1.2.2 To estimate EFS and describe toxicity in patients with newly diagnosed high risk NBL randomized to treatment with an 131I-MIBG-containing Induction prior to BuMel ASCT. 1.2.3 To describe the OS and response rates (evaluated per INRC criteria prior to ASCT and prior to post-Consolidation therapy) for patients with high-risk neuroblastoma treated with or without 131I-MIBG or crizotinib. 1.2.4 To prospectively evaluate the relationship of response rate per revised International Neuroblastoma Response Criteria (INRC) to EFS and OS in patients with high-risk NBL treated with and without the addition of 131I-MIBG or crizotinib. 1.3 Exploratory Aims 1.3.1 To evaluate whole body radiation dose, tumor factors, and host factors as potential predictors of efficacy and/or toxicity associated with 131I-MIBG therapy and transplant conditioning. 1.3.2 To determine whether the efficacy (end-Induction response, EFS, and OS) of crizotinib is associated with specific ALK mutations or ALK amplification. 1.3.3 To characterize changes in tumor markers [circulating tumor DNA, including ALK and other tumor specific genetic aberrations, and circulating GD2] over time in response to protocol therapy. 1.3.4 To correlate results of tumor and host profiling with end-Induction response and EFS. 1.3.5 To prospectively evaluate EFS for patients with MIBG non-avid high-risk NBL compared to patients with MIBG-avid high-risk NBL who are randomized to treatment without 131I-MIBG. 1.3.6 To correlate Curie scores calculated from 131I-MIBG post-treatment scans with end Induction response, EFS and OS. 1.3.7 To describe changes in image defined risk factors (IDRFs) over the course of Induction therapy, with correlation to surgical outcomes and local failure rates following primary tumor resection. 1.3.8 To define patterns of failure at time of first relapse or progression in patients with high risk NBL. 1.3.9 To determine the feasibility of prospectively monitoring adverse events using electronic health records. 1.3.10 To compare local, central, and computer assisted Curie score assignment at baseline and during therapy in patients with MIBG-avid high-risk NBL. 1.3.11 To compare late toxicities (including impaired organ function and secondary tumor occurrence) in patients treated with 131I-MIBG or crizotinib to late toxicities in patients who have not received these therapies.

    View All Details
    • Protocol Number:
      111803

    • Principal Investigator:
      Richard Drachtman M.D.

    • Phase:
      Phase III

    • Scope:
      National

    • Applicable Disease Sites:
      Any Site

      • Contacts:

      • Rutgers University Prinicipal Investigator: Richard Drachtman M.D.

    Read Inclusion & Exclusion Criteria

    Inclusion Criteria:

    • Patients must be enrolled on ANBL00B1 or APEC14B1 prior to enrollment on ANBL1531
    • Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible:
    • Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
    • MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
    • Age > 547 days regardless of biologic features
    • Patients with INRG stage MS disease with MYCN amplification
    • Patients with INRG stage L2 disease with MYCN amplification
    • Patients > 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progressed to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M
    • Patients >= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M
    • Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing); patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible; patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible
    • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows:
    • 1 to < 2 years: male = 0.6; female = 0.6
    • 2 to < 6 years: male = 0.8; female = 0.8
    • 6 to < 10 years: male = 1; female = 1
    • 10 to < 13 years: male = 1.2; female = 1.2
    • 13 to < 16 years: male = 1.5; female = 1.4
    • >= 16 years: male = 1.7; female = 1.4
    • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
    • Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN; for the purposes of this study, ULN for SGPT (ALT) is 45
    • Shortening fraction of >= 27% by echocardiogram, or ejection fraction of > 50% by echocardiogram or radionuclide angiogram
    • No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure

    Exclusion Criteria:

    • Patients with INRG stage L2 tumors without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
    • Patients with bone marrow failure syndromes
    • Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
    • Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential
    • Lactating females who plan to breastfeed their infants
    • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation

    Please note that we have obtained the inclusion and exclusion criteria information from the National Institutes of Health’s clinical trials web site www.clinicaltrials.gov. The listed criteria may not necessarily reflect recent amendments to the protocol and the current criteria.

    For further information about clinical trials, please contact us at 732-235-7356 or 844-CANCERNJ.

    • Newark Beth Israel Medical Center
    • Rutgers Cancer Institute of New Jersey
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