A Study of Avapritinib in Paediatric Patients with Solid Tumours
Research type
Research Study
Full title
A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling
IRAS ID
302854
Contact name
Yolanda Princewill
Contact email
Sponsor organisation
Blueprint Medicines Corporation
Eudract number
2020-005234-15
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 4 months, 1 days
Research summary
This study will be in a Phase 1/2, Single-arm Study to evaluate the Safety, Pharmacokinetics (drug levels), and Anti-tumour Activity of Avapritinib in Paediatric Patients with Solid Tumours dependent on KIT or PDGFRA Signalling. Avapritinib is a type I tyrosine kinase inhibitor (TKI) designed to inhibit strongly and selectively KIT and PDGFRA mutants and Avapritinib has shown potent inhibition and clinical activity in adult cancers that harbour these alterations including mutations not targeted by other approved therapies. In the paediatric population, KIT and PDGFRA mutations have been reported in central nervous system (CNS) tumours (glioblastoma, medulloblastoma and high-grade glioma [HGG] rarely but the most aggressive type of brain tumour, Diffuse Midline Glioma has recently been shown to be highly dependent on PDGFRA signalling and therefore represents an attractive target. Thus, paediatric patients with relapsed solid tumours with mutations in KIT or PDGFRA and patients with progressive gliomas with H2K27M mutations represent a high unmet medical need, for a study of Avapritinib in childhood cancer. Avapritininb is an oral medication take once daily continuously (paediatric formulation available in phase 2) which achieves high levels of active drug in the brain necessary to treat brain tumours. The entire study will enrol a total of 37 patients. The phase I part (approx. 12 patients) will establish a safe dose for the paediatric population and then the phase 2 part (at least 25 patients) will take this dose forward to further assess safety and anti-tumour activity.
REC name
East of England - Essex Research Ethics Committee
REC reference
22/EE/0092
Date of REC Opinion
18 Jul 2022
REC opinion
Further Information Favourable Opinion