Canadian clinical trial registry

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Study Description

This is a study of trametinib (an oral drug called a MEK inhibitor) in children and adolescents who have any of the following types of cancer or tumours:

- patients with neurofibromatosis type 1 (NF1) that have low grade glioma, or
- patients with neurofibromatosis type 1 (NF1) that have plexiform neurofibroma,
- patients with low grade glioma that has a gene change called a BRAF fusion (in the tumour)
- patients with glioma of any grade (low or high) with activation of proteins called the MAPK/ERK pathway.

For all patients (except for those with plexiform neurofibroma), the disease must have recurred or grown in size after a first treatment.

Inclusion Criteria

• Patient must be aged ≥ 1 month (corrected age) to ≤ 25 years at the time of study enrollment
• Participants must belong to one of the groups described in the study description section
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

Publications

Perreault S, Larouche V, Tabori U, Hawkin C, Lippé S, Ellezam B, Décarie JC, Théoret Y, Métras MÉ, Sultan S, Cantin É, Routhier MÈ, Caru M, Legault G, Bouffet É, Lafay-Cousin L, Hukin J, Erker C, Jabado N. A phase 2 study of trametinib for patients with pediatric glioma or plexiform neurofibroma with refractory tumor and activation of the MAPK/ERK pathway: TRAM-01. BMC Cancer. 2019 Dec 27;19(1):1250. doi: 10.1186/s12885-019-6442-2.

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Study Description

The purpose of this study is to determine the best dose, benefits and/or side effects of 2 drugs (magrolimab and dinutuximab) when given in combination to treat neuroblastoma that has come back (relapsed) or does not respond to treatment (refractory), or osteosarcoma that has come back (relapsed). These drugs (magrolimab and dinutuximab) interfere with the ability of cancer cells to grow and spread, using them in treatment may stop or shrink cancer growth in cases of neuroblastoma and osteosarcoma.

An additional aim of this study is determining the safety of these drugs (magrolimab and dinutuximab) when given after surgery to remove tumors from the lungs.

Inclusion Criteria

• Age between 1 and 35 years
• Patients must have:
  • neuroblastoma that has come back (relapsed) or does not respond to treatment (refractory) or
  • osteosarcoma that has come back (relapsed)
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

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Study Description

This is a phase II study to treat patients with neuroblastoma that has come back (relapsed) or is not responding to previous treatment (refractory). This study is seeing if combining eflornithine (a drug used to block the production of chemicals that may cause growth of cancer cells) with drugs used in chemotherapy/immunotherapy (irinotecan hydrochloride, temozolomide, sargramostim and dinutuximab) may work better in treating patients with relapsed or refractory neuroblastoma compared to using irinotecan hydrochloride, temozolomide, sargramostim and dinutuximab alone. Patients will be randomly assigned to 1 of 2 regimens, one regimen will recieve eflornithine with the chemotherapy/immunotherapy drugs and the other regimen will recieve chemotherapy/immunotherapy drugs without eflornithine.

Inclusion Criteria

• Patients must have a neuroblastoma that has come back, progressed or not responded to previous treatment
• Patients must be 1 year of age or older
• Patients must be up and about at least 50% of their waking hours
• 14 days must have passed if patients have recieved any treatment that supresses bone marrow
• >= 21 days must have passed from infusion of antibodies
• Patients must not have received radiation for a minimum of 4 weeks before joining the study. Palliative radiation while on study is not permitted.
• Blood work must come back within the acceptable ranges
• Patients must have no evidence of shortness of breath, and no need for supportive oxygen 
• Patients with a history of central nervous system (CNS) disease must have no evidence of active CNS disease at the time of study enrollment
• Patients with seizure disorders may be enrolled if seizures are well controlled on proper medication
• Patients of childbearing potential must have a negative pregnancy test to be eligible for this study and must agree to use adequate contraception while enrolled on this study.

Other inclusion and exclusion criteria may apply and will be reviewed by your treating team.

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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.

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Study Description

This study is to find the appropriate dose as well as to assess the safety and effectiveness of a drug called quizartinib for children with relapsed or refractory acute myeloid leukemia (AML) with a certain mutation called FLT3-ITD. Quizartinib is a drug designed to specifically block the FLT3 protein when it is abnormal in a leukemia cell.

Inclusion Criteria

• Children 1 month to 21 years of age
• Acute myeloid leukemia that has come back or is not responding to current treatment
• Presence of the FLT3-ITD mutation in the leukemia
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

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Study Description

This clinical trial studies the side effects and how well entrectinib (a medication taken by mouth) works in treating patients with solid tumors and brain tumours with a specific gene change called a "NTRK fusion" or a "ROS1 fusion". Entrectinib may stop the growth of cancer cells with NTRK or ROS1 fusions by blocking the TRK or ROS1 enzymes needed for cell growth.

Inclusion Criteria

• Age up to 18 years
• Solid tumors and brain tumours with a change in a called a NTRK1/2/3 fusion or a ROS1 fusion
• Cancer that has come back (relapse) or is not improving despite treatment (progression)
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

Publications

Doebele RC, Drilon A, Paz-Ares L, Siena S, Shaw AT, Farago AF, Blakely CM, Seto T, Cho BC, Tosi D, Besse B, Chawla SP, Bazhenova L, Krauss JC, Chae YK, Barve M, Garrido-Laguna I, Liu SV, Conkling P, John T, Fakih M, Sigal D, Loong HH, Buchschacher GL Jr, Garrido P, Nieva J, Steuer C, Overbeck TR, Bowles DW, Fox E, Riehl T, Chow-Maneval E, Simmons B, Cui N, Johnson A, Eng S, Wilson TR, Demetri GD; trial investigators. Entrectinib in patients with advanced or metastatic NTRK fusion-positive solid tumours: integrated analysis of three phase 1-2 trials. Lancet Oncol. 2020 Feb;21(2):271-282. doi: 10.1016/S1470-2045(19)30691-6. Epub 2019 Dec 11. Erratum in: Lancet Oncol. 2020 Feb;21(2):e70. Lancet Oncol. 2020 Jul;21(7):e341. Lancet Oncol. 2020 Aug;21(8):e372.

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Study Description

This study is aimed at improving the outcome for children with High Grade Glioma (HGG). Temozolomide, a medication taken by mouth, has been shown to improve survival in adults with glioblastoma, when taken during and after radiation. In this study, patients with a HGG that has specific molecular features (wild-type for H3-K27, IDH and BRAF) will receive experimental therapy of veliparib (given by mouth) during radiation therapy, followed by veliparib in combination with temozolomide.

This study will test whether this combination therapy may improve survival in children with newly diagnosed HGG that are wild-type for H3-K27, IDH and BRAF. Patients with IDH mutant glioma are also eligible. The outcomes of patients on this study will be compared to other patients with clinically and molecularly similar HGG treated with temozolomide alone.

(Retrieved from: https://www.childrensoncologygroup.org/acns1721)

Inclusion Criteria

• Patients must be at least 3 years old and less than 22 years of age. Patients less than 25 years old may be eligible to enroll if they have a specific type of high grade glioma
• Patients must have newly diagnosed High Grade Glioma, that does not include brainstem or spinal cord tumors, or disease that has spread to other parts of the body (metastatic disease)
• Patients must agree to the study activities outlined in the consent forms; including MRI's, blood tests, biopsies etc - as they apply
• Patients must be able to walk and do routine activities for more than half of their normal waking hours
• If the patient has a seizure disorder, they may be enrolled if the seizures are well-controlled
• A written consent form must be signed by the patients and/or their parents/legal guardians

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

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Study Description

The purpose of this study is to test the safety of a cancer drug called repotrectinib in children. The cancer must have a change in a particular gene (ALK, ROS1 or NTRK). Those gene changes lead to abnormal proteins which may cause cancer cells to grow. Repotrectinib blocks the actions of these genes in cancer cells and can therefore be used to treat cancer.

The first study part (Phase 1) is done to determine what dose level of repotrectinib is safe for children, how the drug is absorbed.

The main purpose of the second study part (Phase 2) is to investigate how well and how long different cancer types respond to the treatment with repotrectinib.

This study is divided into 2 stages/phases.

The first stage or phase evaluates the safety and tolerability of the drug (repotrectinib) in patients with advanced cancers. This allows researchers to determine the Maximum Tolerated Dose (MTD) or Maximum Administered Dose (MAD) which can then be used to select the recommended dose for patients.

The second stage or phase will determine the effectiveness of the drug (repotrectinib) in treating advanced cancers.

Inclusion Criteria

For the first phase:

• Age <12 years
• Patients with advanced or metastatic cancer, including brain tumours or certain types of lymphoma
• The tumour must have a change in one of the following genes: ALK, ROS1 or NTRK
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

For the second phase:

• Age 12 to less than 25 years
• Patients with advanced or metastatic cancer, including brain tumours or certain types of lymphoma
• The tumour must have a change in one of the following genes: ALK, ROS1 or NTRK
• Some patients may be eligible even if they have already received another drug blocking NTRK in the past
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.