Official Title
Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST)
Brief Summary

This is a US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.

Approved for marketing
Intermediate-size Population
GIST

Drug: Avapritinib

Avapritinib will be administered orally (PO) at a dose of 300 mg once daily (QD) in continuous 28-day cycles.
Other Name: BLU-285

Eligibility Criteria

Inclusion Criteria:

1. Patient is ≥ 16 years of age.

2. Diagnosis of unresectable or metastatic GIST

3. The patient is not eligible for an ongoing study of avapritinib or cannot access an
ongoing study of avapritinib.

4. Patient has received 3 or more TKI therapies including imatinib, or the patient has
GIST that carries a mutation in exon 18 of the PDGFRA gene (such as D842V).

5. The patient has adequate vital organ function, including heart, lungs, liver, kidneys,
bone marrow and endocrine, and is expected to tolerate therapy with a TKI.

6. Patient or legal guardian, if permitted by local regulatory authorities, provides
informed consent.

Exclusion Criteria:

1. Patients who have poor organ function as defined by one or more of the following
laboratory parameters;

1. Total bilirubin > 2 × ULN; or > 3 × ULN in the presence of Gilbert's Disease;

2. Platelet count < 75 × 10^9/L.

2. Patient requires therapy with a concomitant medication that is a strong inhibitor or
strong inducer of cytochrome P450 (CYP) 3A4

3. Patient has had a major surgical procedure (minor surgical procedures such as central
venous catheter placement, tumor needle biopsy, and feeding tube placement are not
considered major surgical procedures) within 14 days of the first dose of avapritinib.

4. Patient has a history of a cerebrovascular accident or transient ischemic attacks
within 1 year prior to the first dose of program drug.

5. Patient has a known risk of intracranial bleeding, such as a brain aneurysm or history
of subdural or subarachnoid bleeding.

6. Women who are unwilling, if not postmenopausal or surgically sterile, to abstain from
sexual intercourse or employ highly effective contraception from the time of
randomization and for at least 30 days after the last dose of avapritinib. Men who are
unwilling, if not surgically sterile, to abstain from sexual intercourse or employ
highly effective contraception from the time of first dose and for at least 90 days
after the last dose of avapritinib.

7. Women who are pregnant, as documented by a serum beta human chorionic gonadotropin
(β-hCG) pregnancy test consistent with pregnancy, obtained within 7 days before the
randomization. Females with β-hCG values that are within the range for pregnancy but
are not pregnant (false-positives) may be enrolled with written consent of the
Sponsor, after pregnancy has been ruled out. Females of non- childbearing potential
(postmenopausal for more than 1 year; bilateral tubal ligation; bilateral
oophorectomy; hysterectomy) do not require a serum β-hCG test.

8. Women who are breast feeding.

Eligibility Gender
All
Eligibility Age
Minimum: 16 Years ~ Maximum: N/A
Blueprint Medicines Corporation
NCT Number