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| Diagnosis | Solid Tumor, Relapsed Solid Neoplasm, CNS Tumor | Study Status | Closed |
| Phase | I/II |
| Age | 2 Years to 17 Years | Randomisation | NO |
| Line of treatment | Disease relapse or progression |
| Routes of Treatment Administration | Drug: avapritinib (Route: Oral)
Other Name: BLU-285 |
| Last Posted Update | 2024-09-18 |
| ClinicalTrials.gov # | NCT04773782 |
International Sponsor
Blueprint Medicines CorporationPrincipal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel MorgensternCentres
Study Description
This is a phase 1/2 study that studies the drug avapritinib in patients 2-17 years old with solid tumors that have come back (relapsed) or have not responded to previous treatment (refractory). The tumor must have a certain genetic mutation (in KIT, PDGFRA or H3K27M (glioma only)) to qualify you for the study. The study consists of 2 parts; in part 1, more information will be gathered about the drug's safety and how it moves through the body (pharmacokinetics or PK) and in part 2, the effectiveness, safety and PK will continued to be monitored at the recommended dose.
Inclusion Criteria
- Patients must be between 2-17 years old
- Patients must have a diagnosed solid or central nervous system (CNS) tumor that has progressed despite prior standard therapy or no alternative treatment is available
- Patients must be able to be up and about at least half of their waking hours (if applicable)
- Blood work must come back within acceptable ranges within 14 days of the first study dose
- Patients must not be pregnant on the trial, participants of child bearing potential must agree to use approved contraception methods
- Patients and/or their families/caregivers must sign a consent form outlining all assessments and requirements
