Canadian clinical trial registry

Diagnosis	Central nervous system (CNS) HR-EBT: Embryonal Tumor (various), Group 3 and 4 Medulloblastoma, Atypical Teratoid Rhabdoid Tumor, Pineoblastoma, CNS Neuroblastoma, Medulloepithelioma	Study Status	Open
Phase	I
Age	up to (and including) 6 Years old	Randomisation	NO
Line of treatment	First line treatment
Routes of Treatment Administration	Induction Phase: - Drug: Double Therapy (Cytarabine, Hydrocortisone) - intrathecal (IT) - Drug: Cisplatin - intravenous (IV) - Drug: Vincristine - intravenous (IV) - Drug: Etoposide - intravenous (IV) - Drug: Cyclophosphamide - intravenous (IV) - Drug: Mesna - intravenous (IV) - Drug: Filgrastim - subcutaneous or intravenous (SC or IV) Consolidation Phase: - Drug: Carboplatin - intravenous (IV) - Drug: Thiotepa - intravenous (IV) - Drug: Filgrastim - subcutaneous or intravenous (SC or IV) Maintenance Arms (A and/or B): - Drug: Topotecan - intrathecal (IT) - Drug (Maintenance A Only): Tamoxifen - oral (PO) - Drug: ISOtretinoin - oral (PO) - Drug (Maintenance B Only): Celecoxib - oral (PO) - Drug (Maintenance B Only): Temozolomide - oral (PO) - Drug (Maintenance B Only): Cyclophosphamide - oral (PO) - Drug (Maintenance B Only): Etoposide - oral (PO)
Last Posted Update	2026-01-22
ClinicalTrials.gov #	NCT06942039

International Sponsor

C17 Council

Principal Investigators for Canadian Sites

The Hospital for Sick Children - Dr. Annie Huang
CHU Ste. Justine - Dr. Sébastien Perreault
Alberta Children's Hospital - Dr. Lucie Lafay-Cousin
BC Children's Hospital - Dr. George Michaiel
London Children's Hospital - Dr. Shayna Zelcer

Centres

The Hospital for Sick Children

Medical contact

Dr. Daniel Morgenstern

Social worker/patient navigator contact

Karen Fung

Clinical research contact

New Agent and Innovative Therapies (NAIT)

CHU Ste-Justine

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

alexandra.zorzi@lhsc.on.ca

Children's Hospital of Western Ontario

Medical contact

Dr. Alexandra Zorzi

Dr. Shayna Zelcer

shayna.zelcer@lhsc.on.ca

Social worker/patient navigator contact

Cindy Milne Wren

Cindy.MilneWren@lhsc.on.ca

Jessica Mackenzie Harris

Jessica.MacKenzieHarris@lhsc.on.ca

Clinical research contact

Mariam Mikhail

Mariam.mikhail@lhsc.on.ca

Alberta Children's Hospital

Medical contact

Dr. Victor Lewis

victor.lewis@ahs.ca

Social worker/patient navigator contact

Wendy Pelletier

wendy.pelletier@ahs.ca

Clinical research contact

Debra Rich

debra.rich@ahs.ca

BC Children's Hospital

Medical contact

Rebecca Deyell

rdeyell@cw.bc.ca

Social worker/patient navigator contact

Ilana Katz

ilana.katz@cw.bc.ca

Clinical research contact

Hem/Onc/BMT Clinical Trials Unit

CRA@cw.bc.ca

Study Description

A pilot study to see if it is possible and safe to add medicine given into the spinal fluid (intrathecal chemotherapy) and continued treatment (maintenance therapy) after strong chemotherapy for young children under 6 years old who have newly diagnosed high-risk brain tumors.

Inclusion Criteria

Age: Children 6 years old or younger at the time their brain tumor is confirmed.
Tumor types: Certain rare, aggressive brain or spinal tumors, including ATRT, medulloblastoma (group 3 or 4), pineoblastoma, ETMR, and other similar embryonal brain tumors.
MRI scans: MRI of the brain and spine (with and without contrast) must be done before and after surgery.
Lumbar puncture (spinal tap): A sample of spinal fluid is recommended (if safe to do) before or after surgery, but not required.
Must meet all lab and organ function requirements
Must be well enough to 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.

MIRV - A Phase 1/2 Study of Mirdametinib and Vinblastine for Newly Diagnosed or Previously Untreated Patients With Pediatric Low-grade Glioma and Activation of the MAPK Pathway

Open

Diagnosis	Pediatric Low-grade Glioma	Study Status	Open
Phase	I/II
Age	2 Years to 25 Years	Randomisation	NO
Line of treatment	First line treatment
Routes of Treatment Administration	Drug: Mirdametinib (oral) Drug: Vinblastine (IV)
Last Posted Update	2026-01-20
ClinicalTrials.gov #	NCT06666348

International Sponsor

St. Justine's Hospital

Principal Investigators for Canadian Sites

CHU Sainte-Justine - Dr. Sebastian Perreault
The Hospital for Sick Children - Dr. Anthony Liu
London Children's Hospital - Dr. Shayna Zelcer

Centres

CHU Ste-Justine

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

The Hospital for Sick Children

Medical contact

Dr. Daniel Morgenstern

Social worker/patient navigator contact

Karen Fung

Clinical research contact

New Agent and Innovative Therapies (NAIT)

alexandra.zorzi@lhsc.on.ca

Children's Hospital of Western Ontario

Medical contact

Dr. Alexandra Zorzi

Dr. Shayna Zelcer

shayna.zelcer@lhsc.on.ca

Social worker/patient navigator contact

Cindy Milne Wren

Cindy.MilneWren@lhsc.on.ca

Jessica Mackenzie Harris

Jessica.MacKenzieHarris@lhsc.on.ca

Clinical research contact

Mariam Mikhail

Mariam.mikhail@lhsc.on.ca

Study Description

This is a research study combining an oral drug called mirdametinib with an intravenous chemotherapy drug called vinblastine for the treatment of patients with pediatric low grade glioma with certain mutations.

In this first part, doctors will carefully test different dose levels of the two medicines to find the safest and most appropriate dose to use in the next phase. Patients will take mirdametinib by mouth twice a day at a set dose for 13 treatment cycles (each cycle is 28 days). They will also receive vinblastine through an IV once a week for 17 cycles. If side effects happen, the dose can be lowered up to two times to keep treatment safe. After treatment ends, patients will come back for check-ups every 6 months for 3 years. These visits help doctors track how the tumour responds over time and how patients are doing overall.

Inclusion Criteria

A parent or substitute decision maker (or the participant if they have capacity) must sign a consent form before joining the study
Participants need to be able to come to scheduled appointments, take study medicines as instructed, complete lab tests, and have MRIs as part of their care
Participants must be between 2 and 25 years old when starting mirdametinib.
Participants must have a type of low-grade brain tumour (PLGG) with one of the following:
- An NF1 gene change (based on medical criteria or genetic testing), or
- A KIAA1549-BRAF fusion, or
- Another MAPK pathway change, except for the BRAF V600E mutation.
Tumour tissue is needed for testing (usually a stored tissue block, and fresh frozen tissue if available).
- Children with NF1 and low-grade glioma can still join even if they never had surgery or a biopsy.
A recent MRI before starting
Must be well enough to take part in daily activities at least 50% of waking hours
Organ and bone marrow function and body surface area must be within allowable limits
Teens and adults who could become pregnant must use effective birth control during the study
Must be able to swallow the medication by mouth, as this drug cannot be given through feeding tubes

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

ONITT - A Randomized Phase I/II Study of Talazoparib or Temozolomide in Combination With Onivyde in Children With Recurrent Solid Malignancies and Ewing Sarcoma

Closed to enrollment

Diagnosis	Ewing Sarcoma, Hepatoblastoma, Neuroblastoma, Osteosarcoma, Rhabdoid Tumor, Rhabdomyosarcoma, Wilms, Sarcoma	Study Status	Closed to enrollment
Phase	I/II
Age	Child, Adult - (12 Months to 30 Years)	Randomisation	YES
Line of treatment	Disease relapse or progression
Routes of Treatment Administration	Drug: Onivyde (IV) + Drug: Talazoparib (oral) Drug: Onivyde (IV) + Temozolomide: unspecified (oral or IV most likely)
Last Posted Update	2026-01-20
ClinicalTrials.gov #	NCT04901702

International Sponsor

St. Jude Children's Research Hospital

Principal Investigators for Canadian Sites

The Hospital for Sick Children - Dr. Daniel Morgenstern
BC Children's Hospital - Dr. Rebecca Deyell
CHU Ste Justine - Dr Monia Marzouki

Centres

The Hospital for Sick Children

Medical contact

Dr. Daniel Morgenstern

Social worker/patient navigator contact

Karen Fung

Clinical research contact

New Agent and Innovative Therapies (NAIT)

BC Children's Hospital

Medical contact

Rebecca Deyell

rdeyell@cw.bc.ca

Social worker/patient navigator contact

Ilana Katz

ilana.katz@cw.bc.ca

Clinical research contact

Hem/Onc/BMT Clinical Trials Unit

CRA@cw.bc.ca

CHU Ste-Justine

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 study is for children, teens and young adults whose solid tumors have come back or didn’t respond to treatment. It tests two different medicine combinations, and each person is randomly placed into one of two groups. Arm A uses drugs called Onivyde and talazoparib, and Arm B uses Onivyde and temozolomide. The first part of the study focuses on finding the safest doses and has now been completed. Once the safest doses are found, more patients can join “expansion arms” to see how well the treatments work, including groups for those with certain DNA-repair problems. Phase II is now open for patients with Ewing sarcoma, who will also be randomly assigned to Arm A or Arm B to test the same medicine combinations.

Inclusion Criteria

Participants must be > 12 months and <30 years old at the time of enrollment
Must have a type of solid tumor that has come back or not responded to treatment
For Ewing sarcoma patients: the cancer must have come back or not responded to first treatment, and must have a confirmed EWS-related gene change
Must be up and about at least half of waking hours
Must meet all organ function and bloodwork requirements
Enough time must have passed since the last doses of chemo, growth factors, biologics, radiation or other treatments
Anyone able to have children must use effective birth control during the study. Patients who could become pregnant must have a negative pregnancy test confirmed.
Parent/guardian and patient (if assessed to have capacity) must sign consent

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

ARST2031 - A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy With Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) Plus VINO-CPO Maintenance in Patients With High Risk Rhabdomyosarcoma (HR-RMS)

Closed

Diagnosis	Rhabdomyosarcoma	Study Status	Closed
Phase	III
Age	up to 50 Years	Randomisation	YES
Line of treatment	First line treatment
Routes of Treatment Administration	Chemotherapy medications (Cyclophosphamide, Dactinomycin, Vincristine, Vinorelbine), all given intravenously, except for cyclophosphamide that will be taken by mouth too Patients will also receive radiation therapy when participating to this study.
Last Posted Update	2026-01-20
ClinicalTrials.gov #	NCT04994132

International Sponsor

Children's Oncology Group

Principal Investigators for Canadian Sites

The Hospital for Sick Children - Dr. Paul Nathan
Hamilton Health Sciences Centre - Dr. Uma H. Athale
Children’s Hospital of Eastern Ontario (CHEO) - Dr. Donna L. Johnston
Children's Hospital of Western Ontario – Dr. Shayna Zelcer

Centres

The Hospital for Sick Children

Medical contact

Dr. Daniel Morgenstern

Social worker/patient navigator contact

Karen Fung

Clinical research contact

New Agent and Innovative Therapies (NAIT)

nirav.thacker@sickkids.ca

Hamilton Health Sciences Centre, McMaster University

Medical contact

Dr. Carol Portwine

portwc@mcmaster.ca

Social worker/patient navigator contact

Jane Cassano

cassanoj@hhsc.ca

Clinical research contact

Sabrina Millson

sicil@hhsc.ca

Children’s Hospital of Eastern Ontario (CHEO)

Medical contact

Dr. Donna Johnston

djohnston@cheo.on.ca

Dr. Lesleigh Abbott

labbott@cheo.on.ca

Dr. Nirav Thacker

Social worker/patient navigator contact

Sherley Telisma

stelisma@cheo.on.ca

Clinical research contact

Isabelle Laforest

ilaforest@cheo.on.ca

Children's Hospital of Western Ontario

Medical contact

Dr. Alexandra Zorzi

alexandra.zorzi@lhsc.on.ca

Dr. Shayna Zelcer

shayna.zelcer@lhsc.on.ca

Social worker/patient navigator contact

Cindy Milne Wren

Cindy.MilneWren@lhsc.on.ca

Jessica Mackenzie Harris

Jessica.MacKenzieHarris@lhsc.on.ca

Clinical research contact

Mariam Mikhail

Mariam.mikhail@lhsc.on.ca

Study Description

This phase III trial compares the effect of two chemotherapy drug pathways (vinorelbine with vincristine, dactinomycin, and cyclophosphamide (VAC) followed by vinorelbine and cyclophosphamide versus VAC followed by vinorelbine and cyclophosphamide) for the treatment of high risk rhabdomyosarcoma.

Chemotherapy drugs, such as vinorelbine, vincristine, dactinomycin, and cyclophosphamide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Giving vinorelbine and VAC may kill more tumor cells and adding maintenance therapy after VAC therapy may help get rid of the cancer and/or lower the chance that the cancer comes back.

Inclusion Criteria

Patients must be =< 50 years of age
Study is open to all genders
Patients with newly diagnosed rhabdomyosarcoma (RMS) of any subtype, meeting "high risk" criteria
Various bloodwork (bilirubin, creatinine, etc) must be within an acceptable range
Patients must not be pregnant during the duration of the trial
Additional inclusion and exclusion criteria may apply and will be reviewed by the study team

AHEP1531 - Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Closed to enrollment

OPTIMISE - ARM A - Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

Open

Diagnosis	Relapsed/Refractory Solid tumour (+ lymphoma) or CNS tumour	Study Status	Open
Phase	I/II
Age	0 Years to 21 Years	Randomisation	NO
Line of treatment	Disease relapse or progression
Routes of Treatment Administration	Arm A \| Drug: Irinotecan (IV), Temozolomide (Oral), Paxalisib (Oral)
Last Posted Update	2026-01-08
ClinicalTrials.gov #	NCT06208657

International Sponsor

Australian & New Zealand Children's Haematology/Oncology Group

Principal Investigators for Canadian Sites

The Hospital for Sick Children - Dr. Daniel Morgenstern
CHU Sainte. Justine - Dr. Monia Marzouki
BC Children's Hospital - Dr. Rebecca Deyell

Centres

The Hospital for Sick Children

Medical contact

Dr. Daniel Morgenstern

Social worker/patient navigator contact

Karen Fung

Clinical research contact

New Agent and Innovative Therapies (NAIT)

CHU Ste-Justine

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

BC Children's Hospital

Medical contact

Rebecca Deyell

rdeyell@cw.bc.ca

Social worker/patient navigator contact

Ilana Katz

ilana.katz@cw.bc.ca

Clinical research contact

Hem/Onc/BMT Clinical Trials Unit

CRA@cw.bc.ca

Study Description

This study is eligible for STEP-1 funding. Find more information here.

Both Australia (Zero Childhood Cancer) and Canada (PROFYLE) have programs that study the genetics of childhood cancers to help find better treatments. OPTIMISE is a study that matches patients with new treatments based on their tumor’s genetic profile. The study tests different treatment combinations to find the most effective ones for children with cancer.

The information below is for Treatment Arm A which will be combining a drug called paxalisib with conventional chemotherapy in children and adolescents with cancer.

Inclusion Criteria

Patients must have a cancer that hasn't responded to regular treatment or doesn't have a good treatment option
They must be under 21 years old. Patients 21 years and older may be considered with approval if they have a childhood-type cancer
They need to be part of a precision medicine study (like PROFYLE, ZERO or equivalent)
Must meet tumor measurement requirements
Must be up and about at least 50% of patients waking hours
Patients need to be fully recovered from past treatments
Must meet all organ function and bloodwork requirements
Must agree to allowable methods of birth control. Participants may not be pregnant on this study.
Must agree to sign a consent form and to the study activities

Arm A specific:

Cohort A1: Patients must have disease that can seen on a scan.
Cohort A2: Patients must have a mutation in the appropriate genetic pathway (called PI3K/AKT/mTOR) which will be confirmed by a tumour DNA testing
Cohort A3: Patients can join if their tumour shows any genetic change in the pathway above, even if not from the specific mutations from Cohort A2
Adequate cardiac function

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

BCC016 - Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Open

raoul.santiago.1@ulaval.ca

Diagnosis	Medulloblastoma	Study Status	Open
Phase	II
Age	up to 21 Years	Randomisation	NO
Line of treatment	First line treatment, Disease relapse or progression
Routes of Treatment Administration	Drug: Difluoromethylornithine (Oral) Other Names: Eflornithine, DFMO
Last Posted Update	2025-12-04
ClinicalTrials.gov #	NCT04696029

International Sponsor

Giselle Sholler

Principal Investigators for Canadian Sites

CHU Quebec - Dr. Bruno Michon

Centres

CHU de Quebec

Medical contact

Raoul Santiago

Social worker/patient navigator contact

Isabelle Audet

isabelle.audet@chudequebec.ca

Clinical research contact

Barbara Desbiens

barbara.desbiens@chudequebec.ca

Study Description

Participants with a diagnosis of a brain tumour called medulloblastoma (categorized as high risk/very high risk or has not responded to previous treatment) will receive an oral dose of a medication called difluoromethylornithine (DFMO) twice a day while on study.

Inclusion Criteria

Must be 21 years old or younger to participate
Have a confirmed diagnosis of medulloblastoma that has been categorized as high risk/very high risk or has not responded to previous treatment
A disease assessment that includes imaging, lumbar puncture (only if previously positive) and bone marrow aspiration biopsy (only if previously positive) will be done
Must have had enough time pass from any prior therapy (60 days from last dose of conventional chemotherapy and 45 days for high dose chemotherapy + stem cell transplantation)
Participants must be up and about for at least 50% of waking hours
Must meet all requirements for organ function, clinical and laboratory tests
Females of childbearing potential must have a negative pregnancy test and must agree to use an effective birth control method.
Participants or their legal representatives must sign an informed consent

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

BCC015 - Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

Open

Stephanie.Badour@MUHC.MCGILL.CA

Diagnosis	Neuroblastoma	Study Status	Open
Phase	II
Age	Child, Adult - (up to 31 years)	Randomisation	NO
Line of treatment	Disease relapse or progression
Routes of Treatment Administration	Etoposide: oral DFMO: oral
Last Posted Update	2025-12-04
ClinicalTrials.gov #	NCT04301843

International Sponsor

Giselle Sholler

Principal Investigators for Canadian Sites

Montreal Children's Hospital – Dr. Jitka Stankova
CancerCare Manitoba – Dr. Ashley Chopek
CHU Ste-Justine – Dr. Pierre Teira
CHU Sherbrooke - Dr. Josée Brossard
Alberta Children's Hospital – Dr. Melanie Finkbeiner
CHU de Quebec - Dr. Bruno Michon
Janeway Hospital – Dr. Paul Moorehead

Centres

Montreal Children's Hospital

Medical contact

Clinical Research Unit

mch.rho@muhc.mcgill.ca

Social worker/patient navigator contact

Clinical Research Unit

mch.rho@muhc.mcgill.ca

Clinical research contact

Stephanie Badour

CancerCare Manitoba

Medical contact

Dr. Magimairajan Vanan

mivanan@cancercare.mb.ca

Social worker/patient navigator contact

Rhéanne Bisson

rbisson@hsc.mb.ca

Clinical research contact

Rebekah Hiebert

rhiebert5@cancercare.mb.ca

Megan Ridler

mridler@cancercare.mb.ca

Kathy Hjalmarsson

khjalmarsson@cancercare.mb.ca

CHU Ste-Justine

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