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Checkpoint Inhibitor

Nivolumab for Patients with Biallelic Mismatch Repair Deficiency (bMMRD)

Phase 1 & 2
Waitlist Available
Led By Daniel A Morgenstern, MB BChir PhD
Research Sponsored by The Hospital for Sick Children
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years (60 months) from date of enrollment
Awards & highlights

Study 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.

Eligible Conditions
  • Hypermutated Malignancies
  • Patients with Biallelic Mismatch Repair Deficiency (bMMRD)

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years (60 months) from date of enrollment
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years (60 months) from date of enrollment for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Estimating the feasibility of using Nivolumab as a treatment in bMMRD positive, pediatric patients with refractory or recurrent hypermutated malignancies.
To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with recurrent hypermutated malignancies.
To evaluate the objective response rate to Nivolumab in bMMRD positive pediatric patients with refractory hypermutated malignancies
Secondary outcome measures
Number of Participants with Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment
The progression free survival (PFS) of pediatric patients with progressive or recurrent hypermutated malignancies including bMMRD patients treated with Nivolumab.

Side effects data

From 2022 Phase 3 trial • 541 Patients • NCT02041533
57%
Nausea
54%
Anaemia
51%
Fatigue
39%
Decreased appetite
36%
Malignant neoplasm progression
32%
Constipation
31%
Diarrhoea
30%
Cough
29%
Vomiting
29%
Dyspnoea
25%
Oedema peripheral
24%
Back pain
21%
Pyrexia
21%
Neutropenia
19%
Headache
19%
Hypomagnesaemia
18%
Arthralgia
16%
Asthenia
16%
Dizziness
16%
Neutrophil count decreased
15%
Thrombocytopenia
15%
Insomnia
14%
Hyponatraemia
14%
Rash
14%
Weight decreased
14%
Platelet count decreased
13%
Blood creatinine increased
13%
White blood cell count decreased
12%
Hypokalaemia
12%
Pruritus
12%
Abdominal pain
12%
Pain in extremity
11%
Myalgia
11%
Alanine aminotransferase increased
11%
Aspartate aminotransferase increased
10%
Alopecia
10%
Dry skin
10%
Hypoalbuminaemia
10%
Muscular weakness
10%
Chest pain
10%
Dysgeusia
10%
Pneumonia
10%
Productive cough
9%
Abdominal pain upper
9%
Upper respiratory tract infection
9%
Hypothyroidism
9%
Mucosal inflammation
9%
Peripheral sensory neuropathy
8%
Lacrimation increased
8%
Nasopharyngitis
8%
Non-cardiac chest pain
8%
Epistaxis
8%
Haemoptysis
8%
Stomatitis
8%
Dysphonia
7%
Bronchitis
7%
Chills
7%
Hypertension
7%
Hyperkalaemia
7%
Hyperglycaemia
7%
Blood alkaline phosphatase increased
7%
Dehydration
7%
Lymphocyte count decreased
7%
Anxiety
6%
Hypophosphataemia
6%
Leukopenia
6%
Pleural effusion
6%
Neuropathy peripheral
6%
Pneumonitis
6%
Oropharyngeal pain
5%
Rash maculo-papular
5%
Hypotension
5%
Musculoskeletal chest pain
5%
Malaise
5%
Pain
5%
Dry mouth
5%
Urinary tract infection
5%
Dyspepsia
5%
Gamma-glutamyltransferase increased
5%
Depression
5%
Muscle spasms
4%
Fall
4%
Pulmonary embolism
3%
Metastases to central nervous system
3%
Myocardial infarction
3%
Febrile neutropenia
3%
Musculoskeletal pain
3%
Chronic obstructive pulmonary disease
2%
Malignant pleural effusion
2%
Sepsis
2%
General physical health deterioration
2%
Adrenal insufficiency
2%
Atrial fibrillation
2%
Cardiac failure
2%
Embolism
1%
Hypercalcaemia
1%
Neoplasm progression
1%
Small intestinal haemorrhage
1%
Femur fracture
1%
Cancer pain
1%
Confusional state
1%
Pneumothorax
1%
Circulatory collapse
1%
Bone pain
1%
Pericardial effusion malignant
1%
Atrial flutter
1%
Bronchial obstruction
1%
Superior vena cava syndrome
1%
Syncope
1%
Performance status decreased
1%
Pancytopenia
1%
Colitis
1%
Pericardial effusion
1%
Gastrointestinal haemorrhage
1%
Ileus
1%
Small intestinal obstruction
1%
Lung cancer metastatic
1%
Respiratory tract infection
1%
Respiratory failure
1%
Tumour pain
1%
Appendicitis
1%
Skin infection
1%
Ataxia
1%
Seizure
100%
80%
60%
40%
20%
0%
Study treatment Arm
Investigator Choice of Chemotherapy
Post Chemotherapy Optional Nivolumab
Nivolumab

Trial Design

1Treatment groups
Experimental Treatment
Group I: NivolumabExperimental Treatment1 Intervention
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)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Nivolumab
2014
Completed Phase 3
~4750

Find a Location

Who is running the clinical trial?

The Hospital for Sick ChildrenLead Sponsor
686 Previous Clinical Trials
6,944,782 Total Patients Enrolled
Bristol-Myers SquibbIndustry Sponsor
2,638 Previous Clinical Trials
4,128,503 Total Patients Enrolled
Daniel A Morgenstern, MB BChir PhDPrincipal InvestigatorThe Hospital for Sick Children

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers
Daniel Morgenstern 2017. "Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02992964.
~1 spots leftby Apr 2025
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