Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02992964|
Recruitment Status : Recruiting
First Posted : December 14, 2016
Last Update Posted : August 1, 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 :||20 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02992964
|Contact: Nicole Rickerfirstname.lastname@example.org|
|Contact: Ruben Machado||416-813-7654 ext email@example.com|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia||Not yet recruiting|
|Philadelphia, Pennsylvania, United States, 19104|
|Contact: Michael Fisher, MD firstname.lastname@example.org|
|The Hospital for Sick Children||Recruiting|
|Toronto, Ontario, Canada, M5G 1X8|
|Contact: Daniel Morgenstern, MD PhD 416-813-7654 ext 227565 email@example.com|
|Contact: Ruben Machado, CCRP 416-813-7654 ext 203204 firstname.lastname@example.org|
|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 email@example.com|
|Principal Investigator: Rina Dvir, MD|
|Principal Investigator:||Daniel A Morgenstern, MB BChir PhD||The Hospital for Sick Children|