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

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Brief Title

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

Official Title

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

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.
    



Study Type

Expanded Access




Condition

Advanced Systemic Mastocytosis

Intervention

Avapritinib


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug





Eligibility Criteria

        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.
      

Gender

All

Ages

12 Years - N/A


Contacts

, 617-714-6707, [email protected]



Administrative Informations


NCT ID

NCT04714086

Organization ID

BLU-285-EAP-02


Responsible Party

Sponsor

Study Sponsor

Blueprint Medicines Corporation


Study Sponsor

, , 


Verification Date

January 2021