Avapritinib Rollover Study

Phase 4

Recruiting

• Conditions: Mastocytosis, Systemic
• Interventions: Drug: Avapritinib

• Registration Number: NCT06748001
• Lead Sponsor: Blueprint Medicines Corporation

Brief Summary

The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-11-28
• Study First Submitted: 2024-12-19
• Study First Submitted QC: 2024-12-19
• Study First Posted: 2024-12-24
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-09
• Last Update Posted: 2025-04-10
• Primary Completion: 2027-12-31
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• Participated in a Blueprint Medicines sponsored avapritinib clinical study. The Sponsor reserves the right to not roll over patients who have been on drug hold for an extended time period.
• Completed End of Trial / End of Study (EOT/EOS) visit in the parent study and demonstrated compliance with the parent study requirements.
• Continue to clinically benefit from treatment with avapritinib.
• Able to give written informed consent.
• Agree to continue to use highly effective contraception as defined in this protocol.
• Female patients of childbearing potential must have negative highly sensitive serum pregnancy test within 20 days before the first dose of avapritinib. Women of nonchildbearing potential do not require this test.

Exclusion Criteria

• Participant is participating in another interventional study.
• Participant is unwilling or unable to comply with study procedures and study restrictions.
• Participant is breastfeeding.

Other protocol-defined criteria apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Avapritinib	Avapritinib	Participants who have participated in a Blueprint Medicines sponsored study (the parent study) and continue to benefit from avapritinib.

Primary Outcome Measures

Name	Time	Method
Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation	Up to approximately 4 years

Trial Locations

Locations (5)

Guys and St Thomas NHS Foundation Trust - St Thomas Hospital; 🇬🇧 London, United Kingdom

Universitair Ziekenhuis Antwerpen (UZA); 🇧🇪 Edegem, Belgium

A.O.U. San Giovanni di Dio e Ruggi d'Aragona di Salerno; 🇮🇹 Salerno, Italy

Erasmus Medisch Centrum; 🇳🇱 Rotterdam, Netherlands

Oslo University Hospital; 🇳🇴 Oslo, Norway