Canadian clinical trial registry

Go to Health Care Provider version

Centres

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. 

Go to Health Care Provider version

Centres

Study Description

This study uses a drug called Difluoromethylornithine (DFMO) for patients with neuroblastoma in remission to assess the effectiveness of preventing recurrence. The study also looks at the side effects associated with these medication. DFMO is a medication taken by mouth. It is an blocker of ornithine decarboxylase, an enzyme involved with polyamine biosynthesis in the cancer cells, including neuroblastoma cells. 

Inclusion Criteria

• Patients less than 30 years of age, with high-risk neuroblastoma in complete remission. 

Go to Health Care Provider version

Centres

Study Description

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

This is an early-phase clinical trial testing a new kind of treatment called CLIC-2201. It uses a patient’s own immune cells, which are specially modified to better recognize and fight certain types of cancer that affect B cells. This treatment is being offered to patients whose cancer has come back or has not responded to other treatments.

Inclusion Criteria

Cohort A (B cell lymphoma):

• Must be 18 years of age or older of age at time of consent
• You have B cell lymphoma that has come back or hasn’t gotten better with other treatments
• You’ve already tried at least two treatments, or had a stem cell transplant, or CAR-T therapy before
• You meet the necessary organ function and bloodwork requirements 
• You must be up and about at least half of your waking hours 
• If you could become pregnant or get someone pregnant, you agree to use birth control for a year after the treatment

Cohort B (B-ALL):

• Must be between 1 and 21 years old, and you (and/or your parent or guardian) agree to join the study
• You have B-cell acute lymphoblastic leukemia (B-ALL) that has come back or hasn’t gotten better with other treatments.
• You’ve already tried at least two treatments, or had a stem cell transplant, or CAR-T therapy before
• If you had a treatment that targeted CD22, your cancer must still show signs of that marker
• You meet the necessary organ function and bloodwork requirements 
• You must be up and about at least half of your waking hours 
• If you could become pregnant or get someone pregnant, you agree to use birth control for a year after the treatment
• You are okay with a bone marrow biopsy before starting the study

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

Publications

Both Cohorts A and B

• Any uncontrolled or serious active infection at the time of enrolment.
• Active autoimmune disease requiring immunosuppressive therapy within 4 weeks of enrolment.
• Live vaccine ≤6 weeks prior to enrolment
• Active Graft Versus Host Disease (GVHD) requiring systemic immunosuppressive therapy within 4 weeks of enrolment.
• Treatment with any of the following in the specified time period before leukapheresis:
  • Allogeneic HCT within 3 months,
  • Autologous HCT within 3 months,
  • CD19 CAR-T cell infusion within 3 months,
  • Donor lymphocyte infusion (DLI) within 3 months,
  • Bendamustine within the last 6 months,
  • Any investigational agent within 30 days or 5 half-lives (whichever is shorter),
  • Systemic administration of therapeutic dose corticosteroids (>20 mg/day prednisone or equivalent for adults and ≥ 12 mg/m2/day for paediatric participants) within 7 days prior to leukapheresis.
  • Immunosuppressive therapies (i.e., calcineurin inhibitors, methotrexate, mycophenolate, rapamycin) within 4 weeks.
  • Oral chemotherapy agents (i.e., venetoclax) within 5 half-lives. An exception to this is that bruton tyrosine kinase (BTK) inhibitors like ibrutinib can be continued in participants with mantle cell lymphoma throughout the trial period.
• Other concurrent malignancy or a prior malignancy treated within the past 2 years, except carcinoma in situ of the skin or cervix treated with curative intent and with no evidence of active disease.
• Concomitant genetic syndrome associated with bone marrow failure such as Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure or immunodeficiency syndrome.
• Active (confirmed by PCR) hepatitis B or hepatitis C at time of screening confirmed by PCR.
• Any Human Immunodeficiency Virus (HIV) infection at time of screening.
• Hypersensitivity to fludarabine or cyclophosphamide.
• Any allergy to gentamycin or its derivatives
• Pregnant or nursing participants.

Go to Health Care Provider version

Centres

Study Description

The purpose of this study is to learn more about how safe and effective a drug called silmitasertib (an oral pill) is in combination with other FDA approved chemotherapy drugs to treat certain kinds of solid tumours. 

Inclusion Criteria

• Participants must be 30 years or younger
• A confirmed tumor type of Neuroblastoma, Ewing sarcoma, Osteosarcoma, Rhabdomyosarcoma, or Liposarcoma that has come back or not responded to standard treatment
• Doctors must be able to see signs of cancer on a scan or in the bone marrow
• Must have recovered from past treatments, with enough time passed since chemo, radiation, immunotherapy, or transplant
• Must meet all organ function and bloodwork requirements
• Must be up and about for 50% of waking hours
• If patient can become pregnant, a negative pregnancy test and adequate birth control is required
• Signed informed consent

Go to Health Care Provider version

Centres

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. 

Go to Health Care Provider version

Centres

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. 

Go to Health Care Provider version

Centres

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

Go to Health Care Provider version

Centres

Study Description

This trial studies how well cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or liver cancer after surgery. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, 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 combination chemotherapy after surgery may kill more tumor cells.


Inclusion Criteria

• Patients must be newly diagnosed with hepatic malignancies (liver cancers) such as hepatoblastoma or hepatocellular carcinoma
• Patients must be under 30 years of age 
• All patients and/or their parents or legal guardians must sign a written informed consent