Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers
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|ClinicalTrials.gov Identifier: NCT02992964|
Recruitment Status : Recruiting
First Posted : December 14, 2016
Last Update Posted : December 4, 2018
|Condition or disease||Intervention/treatment||Phase|
|Refractory or Recurrent Hypermutated Malignancies Biallelic Mismatch Repair Deficiency (bMMRD) Positive Patients||Drug: Nivolumab||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers|
|Actual Study Start Date :||May 15, 2017|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||September 2021|
Regimen: Nivolumab will be administered every 14 days until disease progression or treatment discontinuation due to unacceptable toxicities. Treatment may extend up to 2 years in patients who show clinical and radiological benefit.
Dose: 3 mg/kg intravenously as a continuous infusion over 60 min (+/-10 min window)
Nivolumab (also referred to as BMS-936558 or MDX1106) is a human monoclonal antibody (HuMAb; immunoglobulin G4 [IgG4]-S228P) that targets the programmed death-1 (PD-1) cluster of differentiation 279 (CD279) cell surface membrane receptor.
Other Name: OPDIVO
- To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with refractory hypermutated malignancies [ Time Frame: 5 years (60 months) from date of enrollment ]Objective response rate will assessed by physical assessments, lab values, disease assessments and adverse events that are related to treatment. Scheduled time-points and the above assessments are detailed further in section "9.1 Study Calendar" of the protocol.
- To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with recurrent hypermutated malignancies. [ Time Frame: 5 years (60 months) from date of enrollment ]Objective response rate will assessed by physical assessments, lab values, disease assessments and adverse events that are related to treatment. Scheduled time-points and the above assessments are detailed further in section "9.1 Study Calendar" of the protocol.
- Estimating the feasibility of using Nivolumab as a treatment in bMMRD positive, pediatric patients with refractory or recurrent hypermutated malignancies. [ Time Frame: 5 years (60 months) from date of enrollment ]Feasibility of treatment will be measured using a patient's disease response assessment. This means using standard RECIST criteria for solid tumours, iRANO/RANO criteria for CNS malignancies and the revised AML International Working Group (IWG) Criteria for haematological malignancies; modified RECIST criteria for immune response may be considered during the time of study.
- The progression free survival (PFS) of pediatric patients with progressive or recurrent hypermutated malignancies including bMMRD patients treated with Nivolumab. [ Time Frame: 5 years (60 months) from date of enrollment ]
- Number of Participants with Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [ Time Frame: 5 years (60 months) from date of enrollment ]These will be assessed by abnormal findings in physical assessments, lab values, disease assessments and adverse events.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02992964
|Contact: Kristine Van Sickle||416-813-7654 ext email@example.com|
|Contact: Ruben Machado||416-813-7654 ext firstname.lastname@example.org|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia||Not yet recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Michael Fisher, MD email@example.com|
|The Hospital for Sick Children||Recruiting|
|Toronto, Ontario, Canada, M5G 1X8|
|Contact: Daniel Morgenstern, MD PhD 416-813-7654 ext 227565 firstname.lastname@example.org|
|Contact: Ruben Machado, CCRP 416-813-7654 ext 203204 email@example.com|
|Sub-Investigator: Uri Tabori, MD|
|Sub-Investigator: Vijay Ramaswamy, MD|
|Sub-Investigator: Annie Huang, MD|
|Principal Investigator: Daniel Morgenstern, MD PhD|
|Institut Gustave Roussy||Not yet recruiting|
|Villejuif, France, 94800|
|Contact: Jacques Grill, MD 33142114211|
|Principal Investigator: Jacques Grill, MD|
|Tel Aviv Sourasky Medical Centre||Not yet recruiting|
|Tel Aviv, Israel, 64239|
|Contact: Rina Dvir, MD 97236973494 firstname.lastname@example.org|
|Principal Investigator: Rina Dvir, MD|
|Principal Investigator:||Daniel A Morgenstern, MB BChir PhD||The Hospital for Sick Children|