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Expanded Access Program (EAP) for Avapritinib in Patients With AdvSM

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04714086
Expanded Access Status : Approved for marketing
First Posted : January 19, 2021
Last Update Posted : October 6, 2021
Information provided by (Responsible Party):
Blueprint Medicines Corporation

Brief Summary:
This is a US, multicenter, open-label expanded access program designed to provide access to avapritinib in eligible patients with AdvSM until such time that avapritinib becomes available through other mechanisms, or the Sponsor chooses to discontinue the program.

Condition or disease Intervention/treatment
Advanced Systemic Mastocytosis Aggressive Systemic Mastocytosis Systemic Mastocytosis With an Associated Hematological Neoplasm Mast Cell Leukemia Drug: Avapritinib

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: Expanded Access Program (EAP) for Avapritinib in Patients With Advanced Systemic Mastocytosis (AdvSM)

Intervention Details:
  • Drug: Avapritinib
    Avapritinib will be administered orally (PO) at a dose of 200 mg daily (QD), continuously in 28-day cycles
    Other Name: BLU-285

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. Patients who are ≥ 15 years of age, or ≥ 12 years of age AND ≥ 35 kg.
  2. Patients must have a diagnosis of advanced systemic mastocytosis (AdvSM) or smoldering systemic mastocytosis (SSM). AdvSM includes the following:

    • Aggressive systemic mastocytosis (ASM)
    • Systemic mastocytosis with an associated hematological neoplasm of non-mast-cell lineage (SM-AHN)
    • Mast cell leukemia (MCL)
  3. Patient or legal guardian, if permitted by local regulatory authorities, provides written informed consent.
  4. Patient is not eligible for an ongoing study of avapritinib or cannot access an ongoing study of avapritinib.

Exclusion Criteria:

  1. Patient meets any of the following laboratory criteria:

    • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3.0 × upper limit of normal (ULN); no restriction if due to suspected liver infiltration by mast cells.
    • Bilirubin > 1.5 × ULN (>3 x ULN in the case of Gilbert's disease); no restriction if due to suspected liver infiltration by mast cells or Gilbert's disease.
    • Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2 or creatinine> 1.5 × ULN.
    • Platelet count < 50,000/μL (within 4 weeks of the first dose of avapritinib) or receiving platelet transfusion(s).
  2. Patient has a history of a cerebrovascular accident or transient ischemic attacks within 1 year before the first dose of avapritinib or any other known risks of intracranial bleeding.
  3. Patient has a known risk or recent history (12 months before the first dose of avapritinib) of intracranial bleeding (e.g., brain aneurysm, concomitant vitamin K antagonist use).
  4. Patient has a primary brain malignancy or metastases to the brain.
  5. Patient has clinically significant, uncontrolled cardiovascular disease, including Grade III or IV congestive heart failure according to the New York Heart Association classification; myocardial infarction or unstable angina within the previous 6 months; clinically significant, uncontrolled arrhythmias; or uncontrolled hypertension.
  6. Female patients who are unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception during the avapritinib administration period 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 during the avapritinib administration period and for at least 90 days after the last dose of avapritinib.
  7. Women who are pregnant or breast feeding.
  8. 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 prior to the first dose of avapritinib.
  9. Hypersensitivity to avapritinib or to any of the excipients.
  10. Patient is participating in another interventional study.

No Contacts or Locations Provided
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Responsible Party: Blueprint Medicines Corporation Identifier: NCT04714086    
Other Study ID Numbers: BLU-285-EAP-02
First Posted: January 19, 2021    Key Record Dates
Last Update Posted: October 6, 2021
Last Verified: October 2021
Additional relevant MeSH terms:
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Mastocytosis, Systemic
Leukemia, Mast-Cell
Hematologic Neoplasms
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Skin Diseases
Immune Complex Diseases
Immune System Diseases
Leukemia, Myeloid, Acute
Leukemia, Myeloid
Neoplasms by Site
Hematologic Diseases