Canadian clinical trial registry

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Study Description

The purpose of this study is to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). NTRK gene changes lead to abnormal proteins called TRK fusion proteins, which may cause cancer cells to grow. Larotrectinib blocks the actions of these NTRK 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 larotrectinib is safe for children, how the drug is absorbed and changed by their bodies and how well the cancer responds to the drug.

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 larotrectinib.

Inclusion Criteria

• Children from birth through 21 years of age
• Patient with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and for which no standard or available systemic curative therapy exists
• Patient must have a malignancy with a documented NTRK gene fusion.
• Patient with a infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer Patients with infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer may enroll into this cohort with documentation of an ETV6 rearrangement or a documented NTRK fusion by next generation sequencing.

Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

Publications

Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Müller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov;30 Suppl 8:viii31-viii35. doi: 10.1093/annonc/mdz382. Epub 2019 Dec 24.

Hong DS, DuBois SG, Kummar S, Farago AF, Albert CM, Rohrberg KS, van Tilburg CM, Nagasubramanian R, Berlin JD, Federman N, Mascarenhas L, Geoerger B, Dowlati A, Pappo AS, Bielack S, Doz F, McDermott R, Patel JD, Schilder RJ, Tahara M, Pfister SM, Witt O, Ladanyi M, Rudzinski ER, Nanda S, Childs BH, Laetsch TW, Hyman DM, Drilon A. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials. Lancet Oncol. 2020 Apr;21(4):531-540. doi: 10.1016/S1470-2045(19)30856-3. Epub 2020 Feb 24.

Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Müller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov 1;30(Suppl_8):viii31-viii35. doi: 10.1093/annonc/mdz382.

DuBois SG, Laetsch TW, Federman N, Turpin BK, Albert CM, Nagasubramanian R, Anderson ME, Davis JL, Qamoos HE, Reynolds ME, Cruickshank S, Cox MC, Hawkins DS, Mascarenhas L, Pappo AS. The use of neoadjuvant larotrectinib in the management of children with locally advanced TRK fusion sarcomas. Cancer. 2018 Nov 1;124(21):4241-4247. doi: 10.1002/cncr.31701. Epub 2018 Sep 11.

Laetsch TW, DuBois SG, Mascarenhas L, Turpin B, Federman N, Albert CM, Nagasubramanian R, Davis JL, Rudzinski E, Feraco AM, Tuch BB, Ebata KT, Reynolds M, Smith S, Cruickshank S, Cox MC, Pappo AS, Hawkins DS. Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study. Lancet Oncol. 2018 May;19(5):705-714. doi: 10.1016/S1470-2045(18)30119-0. Epub 2018 Mar 29. Erratum in: Lancet Oncol. 2018 May;19(5):e229.

Drilon A, Laetsch TW, Kummar S, DuBois SG, Lassen UN, Demetri GD, Nathenson M, Doebele RC, Farago AF, Pappo AS, Turpin B, Dowlati A, Brose MS, Mascarenhas L, Federman N, Berlin J, El-Deiry WS, Baik C, Deeken J, Boni V, Nagasubramanian R, Taylor M, Rudzinski ER, Meric-Bernstam F, Sohal DPS, Ma PC, Raez LE, Hechtman JF, Benayed R, Ladanyi M, Tuch BB, Ebata K, Cruickshank S, Ku NC, Cox MC, Hawkins DS, Hong DS, Hyman DM. Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children. N Engl J Med. 2018 Feb 22;378(8):731-739. doi: 10.1056/NEJMoa1714448.

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Study Description

Philadelphia-like leukemia has specific genetic changes. This study is to assess the dose and effectiveness of an oral medication called ruxolitinib that targets Philadelphia-like leukemia cells. It blocks a protein called JAK. The medication is given in addition to standard therapy for the leukemia. 

Based on the leukemia genetics and initial response to treatment, patients are divided into 4 groups. 

Inclusion Criteria

• Patients between 1 and 21 years of age
• New diagnosis of Philadelphia-like, high-risk B-cell acute lymphoblastic leukemia
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

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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

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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

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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.

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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

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Study Description

The purpose of this study is to evaluate the safety and efficacy of combining Focused Ultrasound (Exablate Model 4000 Type 2.0/2.1) with Doxorubicin therapy for the treatment of DIPG in pediatric patients.

Inclusion Criteria

• Age between 5 and 18 years, inclusive
• Patient diagnosed with DIPG
• Must be between 4-12 weeks from completion of radiation therapy
• Able to attend all study visits
• Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so
• If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable

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

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Study Description

Some neuroblastoma have a specific genetic change or mutation called an ALK aberration.  ALK, or anaplastic lymphoma kinase, has been found in several adult and pediatric cancers.  ALK aberrations are present in about 14% of newly diagnosed patients with high-risk neuroblastoma, and can be found more frequently at the time of relapse.  Lorlatinib is a drug called an ALK inhibitor. It is expected to slow or stop the growth of cancer cells which have the ALK aberration. The aim of this phase I/II study is to evaluate the dose, safety, and tolerability of lorlatinib, including the effect it has on the cancer.  Lorlatinib will be given alone or in combination with chemotherapy in children with refractory, relapsed or progressive neuroblastoma with ALK alterations.

Inclusion Criteria

• Patients must have a diagnosis of neuroblastoma either by histologic verification (looking at a sample of the tumour under a microscope) of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines (HVA/VMA).
• Patients are required to have an activating ALK aberration in their tumor, which is identified through genetic testing.
• Patients must have high risk neuroblastoma. Patients who were initially considered low or intermediate risk, but then reclassified as high risk are also eligible.
• Patients must have at least ONE of the following: 1) Recurrent/progressive disease at any time prior to study enrollment, 2) Refractory disease, 3) Persistent disease

Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.