Avapritinib Rollover Study

NCT06748001 | Recruiting Phase 4 FDA Drug

• 1 other identifier: BLU-285-2404
• Study Type: interventional
• Target: 60
• Locations: 6 countries
• Sites: 7

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.

Conditions

Mastocytosis, Systemic

Keywords

Indolent Systemic Mastocytosis; ISM; Advanced Systemic Mastocytosis; AdvSM; Rollover study

Outcome Measures

Primary Outcomes (1)

• Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation

  Up to approximately 4 years

Study Arms (1)

Avapritinib

EXPERIMENTAL

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

Drug: Avapritinib

Interventions

Avapritinib DRUG

Avapritinib will be administered in accordance with the parent study protocol.

Also known as: BLU-285, AYVAKIT®

Avapritinib

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Blueprint Medicines Corporation: lead

Study Sites (7)

Universitair Ziekenhuis Antwerpen (UZA)

Edegem, 2650, Belgium

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

A.O.U. San Giovanni di Dio e Ruggi d'Aragona di Salerno

Salerno, 84131, Italy

RECRUITING

Universitair Medisch Centrum Groningen

Groningen, 9700, Netherlands

RECRUITING

Erasmus Medisch Centrum

Rotterdam, 3015, Netherlands

RECRUITING

Oslo University Hospital

Oslo, 0424, Norway

RECRUITING

Guys and St Thomas NHS Foundation Trust - St Thomas Hospital

London, SE19RT, United Kingdom

RECRUITING

MeSH Terms

Conditions

Mastocytosis, Systemic

Interventions

avapritinib

Condition Hierarchy (Ancestors)

Mastocytosis; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Mast Cell Activation Disorders; Immune System Diseases

Central Study Contacts

Blueprint Medicines

CONTACT

617-714-6707; MedInfo@blueprintmedicines.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 19, 2024
• First Posted: December 24, 2024
• Study Start: November 28, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: February 9, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); CA (1); NO (1); IT (1); BE (1); NL (2)