Early Access Program for Avapritinib in Patients with Advanced or Metastatic GIST

Sponsor: Blueprint Medicines Corporation

Expanded Access
Approved for Marketing
Sponsor score: 40
Inactive

ClinicalTrials ID:NCT03862885

Provider View

This program provides early access to the drug avapritinib for patients with advanced or metastatic gastrointestinal stromal tumors (GIST) who cannot undergo surgery. It is available until the drug becomes accessible through other means or the program is discontinued.

Patient Parameters

Program Overview

This program is designed to offer patients with advanced or metastatic GIST access to avapritinib, a medication that may help manage their condition. The program is open to patients who meet specific criteria and will continue until avapritinib is available through other channels or the program ends.

Eligibility Criteria

Inclusion Criteria

  • Must be 16 years or older.
  • Diagnosed with GIST that cannot be surgically removed or has spread to other parts of the body.
  • Not eligible for or unable to join an ongoing avapritinib study.
  • Have tried at least three other treatments, including imatinib, or have a specific gene mutation (exon 18 of the PDGFRA gene).
  • Have good overall health, including functioning heart, lungs, liver, kidneys, bone marrow, and endocrine system, and can tolerate treatment with a TKI.
  • Must provide informed consent, or have a legal guardian provide it if allowed by local laws.

Exclusion Criteria

  • Poor organ function, indicated by:
    • High bilirubin levels (more than twice the normal limit, or more than three times in Gilbert's Disease).
    • Low platelet count (less than 75 billion per liter).
  • Need for medications that strongly affect liver enzymes (CYP3A4).
  • Major surgery within 14 days before starting avapritinib (minor procedures like catheter placement or biopsies are okay).
  • History of stroke or mini-strokes within the past year.
  • Known risk of brain bleeding, such as an aneurysm or past brain bleeds.
  • Women who are not postmenopausal or surgically sterile must agree to use effective birth control or abstain from sex during treatment and for 30 days after. Men must agree to use effective birth control or abstain from sex during treatment and for 90 days after.
  • Pregnant women, as confirmed by a pregnancy test, unless a false positive is confirmed and approved by the Sponsor.
  • Women who are breastfeeding.

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