TerminatedPhase 1/2

Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers

Stopped: Withdrawal of drug supply/funding

United States, Australia11 participantsStarted 2017-05-15

Plain-language 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.

Who can participate

Age range12 Months – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Consent/ Assent: Patient and/or Legally Acceptable Representative (LAR; such as a parent or guardian, as applicable) must be willing and able to provide written informed consent/assent for the trial as per local requirements.
✓. Age: patients must be ≥ 12 months and \<25 years of age at time of Part I enrollment. Local centres are only obligated to treat/ admit patients in accordance their age range capabilities.
✓. Recurrent or relapse paediatric cancer patients suspected to be hypermutant, including those exhibiting evidence of one or more of the following:
✓. high microsatellite instability (MSI-H) in current or previous tumour;
✓. a mutation causing loss of mismatch repair gene (MLH1, MSH2, MSH6, PMS2, EPCAM or MSH3) expression;
✓. hypermutation by local sequencing in current or previous tumour;
✓. a history of CMMRD, Lynch syndrome, xeroderma pigmentosum (XP), or other established disorder affiliated with an elevated hypermutation rate;
✓. a functional mutation of polymerase genes (POLE or POLD1) in current or previous tumour;

Exclusion criteria

What they're measuring

To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with refractory hypermutated malignancies

Timeframe: 5 years (60 months) from date of enrollment

To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with recurrent hypermutated malignancies.

Timeframe: 5 years (60 months) from date of enrollment

Estimating the feasibility of using Nivolumab as a treatment in bMMRD positive, pediatric patients with refractory or recurrent hypermutated malignancies.

Timeframe: 5 years (60 months) from date of enrollment

Trial details

NCT IDNCT02992964

SponsorThe Hospital for Sick Children

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-11-20

View on ClinicalTrials.gov More Refractory or Recurrent Hypermutated Malignancies trials

Plain-language summary

Who can participate

Age range12 Months – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Consent/ Assent: Patient and/or Legally Acceptable Representative (LAR; such as a parent or guardian, as applicable) must be willing and able to provide written informed consent/assent for the trial as per local requirements.
✓. Age: patients must be ≥ 12 months and \<25 years of age at time of Part I enrollment. Local centres are only obligated to treat/ admit patients in accordance their age range capabilities.
✓. Recurrent or relapse paediatric cancer patients suspected to be hypermutant, including those exhibiting evidence of one or more of the following:
✓. high microsatellite instability (MSI-H) in current or previous tumour;
✓. a mutation causing loss of mismatch repair gene (MLH1, MSH2, MSH6, PMS2, EPCAM or MSH3) expression;
✓. hypermutation by local sequencing in current or previous tumour;
✓. a history of CMMRD, Lynch syndrome, xeroderma pigmentosum (XP), or other established disorder affiliated with an elevated hypermutation rate;
✓. a functional mutation of polymerase genes (POLE or POLD1) in current or previous tumour;

Exclusion criteria

What they're measuring

To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with refractory hypermutated malignancies

Timeframe: 5 years (60 months) from date of enrollment

To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with recurrent hypermutated malignancies.

Timeframe: 5 years (60 months) from date of enrollment

Estimating the feasibility of using Nivolumab as a treatment in bMMRD positive, pediatric patients with refractory or recurrent hypermutated malignancies.

Timeframe: 5 years (60 months) from date of enrollment