Platzhalter Bild KiTZ

KiTZ Clinical Trial Group

Head: Prof. Dr. med. Olaf Witt
Deputy head: Prof. Dr. med. Stefan Pfister

Im Neuenheimer Feld 430
D-69120 Heidelberg

Avapritinib in Tumors with KIT or PDGFRA alterations or Glioma with H3K27M mutations

“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 (BLU-285-3101)”

This is a Phase 1/2, multicenter, open-label trial of avapritinib in patients aged 2 to less than 18 years of age with with relapsed/refractory (R/R) tumors with mutations (including non-synonymous point mutations, insertions, and deletions) in KIT or PDGFRA, or gliomas with the H3K27M mutation, and no available alternative treatment options. This is a single-arm trial in which all patients will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).

Avapritinib tablets are administered orally daily for 28 day cycles.

Inclusion of patients in Heidelberg has been possible since 01 September 2022.


  • Recurrent or refractory  tumor with a in KIT or PDGFRA
  • H3K27M mutant glioma
  • Age 2 - 18 years old
  • Karnofsky-/Lansky-Score > 50


For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (

See NCT04773782.

EudraCT-Number:  2020-005234-15 (Clinical Trials Register)