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Information is also accessible through the patient and families tab. Family friendly summaries are created and reviewed by our advocacy partners. The information is updated to the best of our knowledge but might not reflect the latest information. Note that most studies are only available at a limited number of sites, please click on ‘further information’ for details. Studies, particularly early phase trials, may also temporarily close to enrolment or not have slots available for all treatment groups. In all cases, study teams at individual C17 centres will have the most up-to-date information.

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PBTC-045 - A Safety and Preliminary Efficacy Trial of Pembrolizumab (MK-3475) in Children With Recurrent, Progressive or Refractory Diffuse Intrinsic Pontine Glioma (DIPG), Non-Brainstem High-Grade Gliomas (NB-HGG), Ependymoma, Medulloblastoma or Hypermutated Brain Tumors

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PBTC-045 - A Safety and Preliminary Efficacy Trial of Pembrolizumab (MK-3475) in Children With Recurrent, Progressive or Refractory Diffuse Intrinsic Pontine Glioma (DIPG), Non-Brainstem High-Grade Gliomas (NB-HGG), Ependymoma, Medulloblastoma or Hypermutated Brain Tumors

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DiagnosisHigh-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma, MedulloblastomaStudy StatusOpen
PhaseI
Age1 Year to 29 Years RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Pembrolizumab (IV over 30 minutes)
Last Posted Update2024-02-13
ClinicalTrials.gov #NCT02359565
International Sponsor
National Cancer Institute (NCI)
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Vijay Ramaswamy
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

 

 

Study Description

The purpose of this research study is to investigate an experimental drug, MK-3475 (also called pembrolizumab), in young patients. The drug MK-3475 has been previously studied in adults, in a variety of tumors, but is not yet FDA approved. The first phase is a safety study to assess side effects and best dose of pembrolizumab, the second phase is the efficacy portion to see how well it works in treating childhood brain tumors that have come back (recurrent), progressed, or not responded (refractory) to previous treatment. 

(transcribed from PBTC-045 Summary for Patients and Families)

Inclusion Criteria
  • Patient must have one of the following diagnoses that have come back, progressed or not responded to treatment, to be eligible: 
    • Stratum A : DIPG - currently closed to enrollment
    • Stratum B : Non-brainstem high-grade glioma (NB-HGG)
    • Stratum C : Hypermutated brain tumor
    • Stratum D : Ependymoma
    • Stratum E : Medulloblastoma 
  • Patient must have been treated with prior radiation therapy and/or chemotherapy (exclusions apply)
  • Both males and females of all races and ethnic groups are eligible for this study
  • Patient must be able to be up and about more than 50% of their waking hours
  • Age Requirements:
    • Stratum A  -  1 year to 18 years old during safety portion, <22 years during efficacy, currently closed to enrollment
    • Stratum B  - 1 year to 18 years old during safety portion, <22 years during efficacy
    • Stratum C  - Younger than 30 years old
    • Stratum D  - 1 year to 18 years old during safety portion, <22 years during efficacy
    • Stratum E  - 1 year to 18 years old during safety portion, <22 years during efficacy
  • Patients must have satisfactory blood work prior to enrolling
  • Patient must not have any shortness of breath
  • Human immunodeficiency virus (HIV)-infected patients with undetectable viral load within 6 months are eligible for this trial
  • Patients of child bearing potential must NOT be pregnant, and must agree to use 2 methods of birth control, be surgically sterile, or abstain from heterosexual activity while on study and 6 months after
  • Patient and/or guardian must sign a written consent document

Additional inclusion and exclusion criteria may apply.