Recruiting

Avapritinib

Sponsor:

Blueprint Medicines Corporation

Code:

NCT06748001

Conditions

Mastocytosis, Systemic

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Avapritinib

Study Details

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

Study ID

NCT06748001

Start date

Nov 28, 2024

Status verified date

Feb, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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

Enrollment

60 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Avapritinib

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

Interventions

Avapritinib

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

Primary outcome measure

  • Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation [ Time Frame: Up to approximately 4 years ]

Central Contacts and Locations

Central contacts

Blueprint Medicines

617-714-6707MedInfo@blueprintmedicines.com

Locations

The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada, M5G 1X8

More Information

Sponsor

Blueprint Medicines Corporation

Last update posted

Feb 9, 2026

Last verified

Feb, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Blueprint Medicines Corporation on 2026-02-09.