Registre canadien des essais cliniques

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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.

116 results found

Title
Status

 

PBTC-059 - Phase 1 Trial of Autologous HER2-specific CAR T Cells in Pediatric Patients With Refractory or Recurrent Ependymoma

Closed to enrollment

PBTC-059 - Phase 1 Trial of Autologous HER2-specific CAR T Cells in Pediatric Patients With Refractory or Recurrent Ependymoma

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DiagnosisEpendymomaStudy StatusClosed to enrollment
PhaseI
Age1 Year to 22 YearsRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationBiological: HER2 Specific CAR T Cell (IV) Phase 1 Arm: Patients receive lymphodepletion chemotherapy with cyclophosphamide IV daily on Days -7 to -6 and fludarabine IV daily on Days -5 to -1. Patients receive HER2 CAR T cells IV on Day 0. Treatment repeats every 8 to 12 weeks for 2 additional cycles in the absence of disease progression or unacceptable toxicity. Surgical Arm: Patients receive lymphodepletion chemotherapy with cyclophosphamide IV daily on Days -7 to -6 and fludarabine IV daily on Days -5 to -1. Patients receive HER2 CAR T cells IV on Day 0 followed by surgical tumor resection 4-6 weeks following HER2 CAR T cell infusion. Treatment repeats every 8 to 15 weeks for 2 additional cycles in the absence of disease progression or unacceptable toxicity.
Last Posted Update2025-09-02
ClinicalTrials.gov #NCT04903080
International Sponsor
Pediatric Brain Tumor Consortium
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Joerg Krueger
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

This study evaluates the safety of a type of treatment called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells). In addition to looking for side effects, we will study how well this treatment works against a brain tumor called ependymoma that has come back after treatment (recurrent) or has not responded well to treatment (progressive) in children. The HER2 CAR T cells used in this trial are made from the patient's own blood.

A new gene, called the HER2 CAR, will be inserted into patient's cells to allow them to recognize a protein on the tumor called HER2. These HER2-specific CAR T cells may be able to target and kill ependymoma tumors. This research is also studying how doable it is to provide this type of CAR T cell treatment to children being treated at different hospitals.

Inclusion Criteria
  • Participants must have a diagnosis of ependymoma that is has come back or progressed.
  • Patient must be ≥ 1 but ≤ 22 years of age at the time of enrollment for treatment.
  • Participants must be up and about for 60% of their waking hours
  • Patients must have received last dose of previous chemotherapy at least 21 days before enrollment.
  • Patient must meet all organ function, bone marrow function and laboratory criteria
  • The patient or parent/guardian can understand the consent and is willing to sign a written informed consent document according to institutional guidelines. Age- and developmentally appropriate assent should be obtained as required by institutional guidelines.

Additional inclusion and exclusion criteria applies and will be discussed with you by the study team.

LCH 20C05 - A Phase I Clinical Trial of Neo-antigen Heat Shock Protein Vaccine (rHSC-DIPGVax) in Combination With Checkpoint Blockade for the Treatment of Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma in Childhood

Open

LCH 20C05 - A Phase I Clinical Trial of Neo-antigen Heat Shock Protein Vaccine (rHSC-DIPGVax) in Combination With Checkpoint Blockade for the Treatment of Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma in Childhood

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DiagnosisDiffuse Intrinsic Pontine Glioma (DIPG), Diffuse Midline Glioma, H3 K27M-MutantStudy StatusOpen
PhaseI
Age12 Months to 18 YearsRandomisationNO
Line of treatmentFirst line treatment
Routes of Treatment AdministrationBiological: rHSC-DIPGVax (vaccine) Drug: Balstilimab Drug: Zalifrelimab
Last Posted Update2025-08-27
ClinicalTrials.gov #NCT04943848
International Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Principal Investigators for Canadian Sites
Alberta Children's Hospital - Dr. Aru Narendran
Centres
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich

 

 

Study Description

This study is testing a new treatment for children with DIPG or DMG brain tumors who have recently finished radiation. The treatment combines a vaccine (rHSC-DIPGVax) with two medicines, BALSTILIMAB and ZALIFRELIMAB. The main goal is to see if this combination is safe and can be tolerated. Patients may join if they were diagnosed with DIPG or DMG and completed radiation about 6–10 weeks before entering the study.

Inclusion Criteria
  • Participants must be 12 months - 18 years old with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG) 
  • Must have measurable tumor on scans
  • Body size (BSA) must be at least 0.35 m²
  • Must be up and about at least 50% of waking hours 

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.

PEPN2121 - A Phase 1/2 Study of Tiragolumab (NSC# 827799) and Atezolizumab (NSC# 783608) in Patients With Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors

Closed

PEPN2121 - A Phase 1/2 Study of Tiragolumab (NSC# 827799) and Atezolizumab (NSC# 783608) in Patients With Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors

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DiagnosisRecurrent/Refractory Atypical Teratoid/Rhabdoid Tumor, Kidney Medullary Carcinoma, Malignant Solid Neoplasm, Poorly Differentiated ChordomaStudy StatusClosed
PhaseI/II
Age12 months of age or older RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationAtezolizumab and tiragolumab are given intravenously (IV) as infusions
Last Posted Update2025-07-17
ClinicalTrials.gov #NCT05286801
International Sponsor
National Cancer Institute (NCI)
Principal Investigators for Canadian Sites
CHU Ste Justine - Dr. Monia Marzouki
The Hospital for Sick Children - Dr. Daniel Morgenstern
Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
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

 

This is a phase I/II trial that studies how well the treatments tiragolumab and atezolizumab work when given to children with tumours that have either come back (relapsed) or did not respond to therapy (refractory). These tumours must have a certain mutation, meaning the cancer cells are missing important genes called SMARCB1 and SMARCA4. Tiragolumab and atezolizumab may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

Inclusion Criteria

Part A is now completed, Part B criteria now applies: 

  • Participants must be between 1-18 years old for part A of this study. There is no upper age limit for part B
  • Participants must have one of the following cancers, that has either come back (relapsed) or did not respond to previous treatment (refractory):
    • Renal medullary carcinoma
    • Malignant rhabdoid tumor (extra-CNS)
    • Atypical teratoid rhabdoid tumor (CNS)
    • Poorly differentiated chordoma
    • Epithelioid sarcoma
  • Cancer must have the eligible mutation (SMARCB1 or SMARCA4)
  • Participants must be up and about for at least half their waking hours
  • Participants must meet all bloodwork criteria outlined for this study

Other inclusion and exclusion criteria may apply and will be discussed with you by your clinical team

D0816C00025 - A Phase I, Open-label, Parallel Group Study to Investigate Olaparib Safety and Tolerability, Efficacy and Pharmacokinetics in Paediatric Patients With Solid Tumours

Closed to enrollment

D0816C00025 - A Phase I, Open-label, Parallel Group Study to Investigate Olaparib Safety and Tolerability, Efficacy and Pharmacokinetics in Paediatric Patients With Solid Tumours

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DiagnosisSolid TumoursStudy StatusClosed to enrollment
PhaseI
Age6 Months to 18 Years RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Olaparib (Oral)
Last Posted Update2025-07-16
ClinicalTrials.gov #NCT04236414
International Sponsor
AstraZeneca
Principal Investigators for Canadian Sites
Montreal Children's Hospital - Dr. Sharon Abish
Centres
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 

 

 

Study Description

This is a study to find out whether a drug called olaparib (also called a PARP inhibitor) is safe and well tolerated when administered to children and adolescents with solid tumours or brain tumours, when they have come back or are not improving with treatment.

Inclusion Criteria
  • Patients are ≥ 6 months to <18 years of age 
  • Patients have a solid tumour or brain tumour that has come back (relapsed) or is not improving with treatment (refractory)  
  • There is a gene change in the tumour or in the normal cells of the patient that can be called "homologous recombination repair gene mutation"
  • The patient can swallow tablets
  • Multiple other inclusion criteria could apply and will be reviewed by your treating team.

DAY101-001 (FIREFLY-1) - FIREFLY-1: A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients With BRAF-Altered, Recurrent or Progressive Low-Grade Glioma

Open

DAY101-001 (FIREFLY-1) - FIREFLY-1: A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients With BRAF-Altered, Recurrent or Progressive Low-Grade Glioma

Go to Health Care Provider version

DiagnosisLow-grade glioma or solid tumors with RAF alterationsStudy StatusOpen
PhaseII
AgeChild, Adult - (6 Months to 25 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDay101: oral (tablet formulation)
Last Posted Update2025-07-16
ClinicalTrials.gov #NCT04775485
International Sponsor
Day One Biopharmaceuticals, Inc.
Principal Investigators for Canadian Sites
Montreal Children's Hospital – Dr. Christina Coleman
CHU Ste-Justine – Dr. Sebastien Perreault
CHU de Quebec – Dr. Valerie Larouche

Centres
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Raoul Santiago
 
Social worker/patient navigator contact
Isabelle Audet
 
Clinical research contact
Barbara Desbiens
 

 

 

 

Study Description

The purpose of this study is to see if the experimental drug DAY101 is safe and effective in treating recurrent or progressive (tumor that has come back or isn’t improving despite treatment) glioma or solid tumors in pediatric, adolescent and young adult patients. The drug will be given in the form of oral tablets.

Inclusion Criteria
  • Age between 6 months to 25 years
  • Patient must be diagnosed with low grade glioma or solid tumor, that isn’t improving despite treatment 
  • The tumour must display a genetic change called a "RAF alteration"
  • Patient must be able to swallow oral tablets
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

AALL2121 - A Phase 2 Study of Revumenib (SNDX-5613) in Combination With Chemotherapy for Patients With Relapsed or Refractory KMT2A-Rearranged Infant Leukemia

Open

AALL2121 - A Phase 2 Study of Revumenib (SNDX-5613) in Combination With Chemotherapy for Patients With Relapsed or Refractory KMT2A-Rearranged Infant Leukemia

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DiagnosisKMT2A-Rearranged Infant LeukemiaStudy StatusOpen
PhaseII
Age1 Month to 6 Years RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Revumenib (SNDX-5613) - Oral (or NG/NJ/ND/G-tube) Combination Chemotherapy Regimens: (1) vincristine (IV), prednisone or prednisolone (PO or via NG, ND, NJ, or G-tube), calaspargase pegol-mknl (IV), methotrexate (MTX) (IT), hydrocortisone (IT), cytarabine (IT) (2) fludarabine (IV), high-dose cytarabine (IV) (3) fludarabine (IV), high-dose cytarabine (IV), MTX (IT), hydrocortisone (IT), and cytarabine (IT) (4) monotherapy. Patients may also receive MTX (IT), hydrocortisone (IT), and cytarabine (IT) as clinically indicated.
Last Posted Update2025-07-16
ClinicalTrials.gov #NCT05761171
International Sponsor
Children's Oncology Group
Principal Investigators for Canadian Sites
CHU Ste Justine - Dr. Thai Tran
BC Children's Hospital - Dr. Rebecca Deyell
CancerCare Manitoba - Dr. Stephanie Villeneuve
IWK Health Centre - Dr. Craig Erker
McMaster Children's Hospital - Dr. Uma Athale
London Children's Hospital - Dr. Shayna Zelcer
CHEO - Dr. Donna Johnston
The Hospital for Sick Children - Dr. Jim Whitlock
Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Magimairajan Vanan
Social worker/patient navigator contact
Rhéanne Bisson
 
Clinical research contact
Rebekah Hiebert
Megan Ridler
Kathy Hjalmarsson

 

 

Medical contact
Dr. Craig Erker
Dr. Conrad Fernandez 
Dr. Ketan Kulkarni 
 
Social worker/patient navigator contact
Rhonda Brophy
 
Clinical research contact
Tina Bocking
 
Medical contact
Dr. Carol Portwine
 
Social worker/patient navigator contact
Jane Cassano 
 
Clinical research contact
Sabrina Millson
 
 
Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
 
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
 
Clinical research contact
Mariam Mikhail
Medical contact
Dr. Donna Johnston
 
Dr. Lesleigh Abbott
 
Dr. Nirav Thacker
 
Social worker/patient navigator contact
Sherley Telisma
 
Clinical research contact
Doaa Abdelfattah
 
Isabelle Laforest
 
 
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

This study is for babies and young children with leukemia that has come back or isn’t getting better with treatment. Some kids with leukemia have a gene change called KMT2A rearrangement, which makes the cancer harder to treat and more likely to come back. Doctors are testing a new medicine called revumenib. It is a pill that helps target and kill leukemia cells with this gene change.

In this study, children will get revumenib along with regular chemotherapy. 

Inclusion Criteria
  • Between 1 month to 6 years old
  • First diagnosed with leukemia before age 2
  • Has a certain type of leukemia with a KMT2A gene change that has come back or didn’t respond to treatment
  • Types of leukemia allowed:
    • Acute lymphoblastic leukemia (ALL)
    • Mixed or unclear type (MPAL or ALAL)
  • Up and about at least 50% of waking hours 
  • Can take medicine by mouth or feeding tube
  • Has recovered from any side effects of past treatment
  • Meets all organ function and blood work requirements

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team. 

CFZ008 - Phase 1b Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia

Closed

CFZ008 - Phase 1b Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia

Go to Health Care Provider version

DiagnosisAcute Lymphoblastic LeukemiaStudy StatusClosed
PhaseI
AgeChild, Adult - (1 Year to 21 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment Administrationintravenous (carfilzomib); Other drugs as usually administered for leukemia therapy
Last Posted Update2025-07-16
ClinicalTrials.gov #NCT02303821
International Sponsor
Amgen
Principal Investigators for Canadian Sites
CHU Ste-Justine - Dr. Henrique Bittencourt

Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

This phase I/II trial is trying to determine how much carfilzomib (an anti-cancer drug) should be used, alone and in combination with induction chemotherapy, to treat children with acute lymphoblastic leukemia that has come back (relapsed) or is refractory to treatment. Carfilzomib is an anti-cancer medication called proteasome inhibitor and is given intravenously.

 

Inclusion Criteria
  • Age 1 year to 21 years
  • Acute lymphoblastic leukaemia that has come back (relapsed) or is refractory to treatment
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

M20-429 - A Single Arm, Open-Label, Phase 1b Trial of Epcoritamab in Pediatric Patients With Relapsed/Refractory Aggressive Mature B-cell Neoplasms

Closed

M20-429 - A Single Arm, Open-Label, Phase 1b Trial of Epcoritamab in Pediatric Patients With Relapsed/Refractory Aggressive Mature B-cell Neoplasms

Go to Health Care Provider version

DiagnosisNon-hodgkin LymphomaStudy StatusClosed
PhaseI
Age1 to 25 Years OldRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Epcoritamab Subcutaneous Injection (SC) Other Name: ABBV-GMAB-3013
Last Posted Update2025-07-16
ClinicalTrials.gov #NCT05206357
International Sponsor
AbbVie
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Sarah Alexander
CHU Sainte-Justine - Dr. Henrique Bittencourt
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

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

The purpose of this study is to assess the safety and tolerability of the study drug epcoritamab in pediatric participants with relapsed/refractory aggressive mature B-cell neoplasms. The study drug will be given subcutaneously in 28 day cycles and participants will be followed for a minimum of 3 years after enrolling.

Inclusion Criteria
  • Participants must have Burkitt's or Burkitt-like lymphoma/leukemia, diffuse large B-cell lymphoma, or other aggressive mature B-cell lymphomas 
  • Tumour must have come back (relapsed) or progressed after previous treatment
  • Between 1-18 years of age
  • Participants must be able to be up and about at least half of their waking hours
  • Adequate organ function 

Other inclusion or exclusion criteria may apply and will be discussed with you by the clinical team