Clinical Trial: Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers

Brief Summary: This is an open-label, single arm, multi-center, pilot study of Nivolumab in pediatric patients with recurrent or refractory hypermutant malignancies aged 12 months to 18 years of age. This study is to assess clinical and radiological benefits of treatment with Nivolumab in children with hypermutated cancers, including those with bMMRD syndrome. It is our expectation that patients with bMMRD syndrome will account for the majority of patients enrolled on this study.

Detailed Summary:
Sponsor: The Hospital for Sick Children

Current Primary Outcome:

  • 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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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.


Original Secondary Outcome: Same as current

Information By: The Hospital for Sick Children

Dates:
Date Received: December 2, 2016
Date Started: April 2017
Date Completion: September 2021
Last Updated: April 4, 2017
Last Verified: April 2017