Observational study evaluating the long-term safety and efficacy of avapritinib in the first-line treatment of patients with platelet-derived growth factor receptor alpha (PDGFRA) D842V-mutated gastrointestinal stromal tumour (GIST)

Study identification

• EU PAS number:
  EUPAS41969
• Study ID:
  43762
• Official title and acronym:
  Observational study evaluating the long-term safety and efficacy of avapritinib in the first-line treatment of patients with platelet-derived growth factor receptor alpha (PDGFRA) D842V-mutated gastrointestinal stromal tumour (GIST)
• DARWIN EU® study:
  No
• Study countries:
  Italy

  Netherlands

  Spain

  United States
• Study description:
  This study protocol concerns a non-interventional observational study evaluating the long-term safety (primary objective) and efficacy (secondary objective) of avapritinib in the first-line treatment of patients with platelet-derived growth factor receptor alpha (PDGFRA) D842V mutated gastrointestinal stromal tumour (GIST), (or following ≤4 months of imatinib treatment). This study is an imposed PASS (category 2) as part of the CMA of avapritinib.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Blueprint Medicines

Study timelines

Date when funding contract was signed

Planned:31/08/2021

Actual:31/08/2021

Study start date

Planned:31/12/2022

Actual:31/03/2023

Data analysis start date

Planned:15/10/2026

Date of interim report, if expected

Planned:28/02/2025

Actual:10/02/2025

Date of final study report

Planned:31/03/2027

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Blueprint Medicines Netherlands B.V.

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)
• Regulatory procedure number:
  EMEA/H/C/PSP/S/0092.1