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(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies | Clinical Trials | Clareo Health

COMPLETEDPHASE1

(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies

A Phase 1 Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies

NCT02561988•Blueprint Medicines Corporation

View on ClinicalTrials.gov

Summary

This is a Phase 1, open-label, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antineoplastic activity of avapritinib (also known as BLU-285), administered orally (PO), in adult patients with advanced systemic mastocytosis and other relapsed or refractory myeloid malignancies. The study consists of 2 parts:, dose-escalation (Part 1) and expansion (Part 2).

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•For Part 1:Patients must have one of the following diagnoses based on World Heath Organization (WHO) diagnostic criteria:
•Aggressive systemic mastocytosis (ASM).
•Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) and at least 1 C-finding attributable to systemic mastocytosis (SM). The AHN must be myeloid, with the following exceptions that are excluded: Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS) that is very high- or high-risk as defined by the International prognostic scoring system for myelodysplastic syndromes (IPSS-R) and Philadelphia chromosome positive malignancies.
•Mast cell leukemia (MCL).
•Histologically- or cytologically- confirmed myeloid malignancy that is relapsed or refractory to standard treatments. AML, MDS that is very high- or high-risk as defined by the IPSS-R, and Philadelphia chromosome positive malignancies are excluded.
•Upon discussion with the sponsor, other relapsed or refractory, potentially avapritinib-responsive hematologic neoplasms (e.g., evidence of aberrant KIT or platelet derived growth factor receptor (PDGFR) signaling) may be considered for enrollment.
•For Part 2, patients must have one of the following diagnoses, based on WHO diagnostic criteria:
•ASM.
•SM-AHN. The AHN must be myeloid, with the following exceptions that are excluded: AML, MDS that is very high- or high-risk as defined by the IPSS-R, and Philadelphia chromosome positive malignancies.
•MCL.
+8 more criteria

Exclusion Criteria

•QT interval corrected using Fridericia's formula (QTcF) \>480 milliseconds
•Platelet count \<50,000/μL (within 4 weeks of the first dose of study drug) or receiving platelet transfusion(s)
•Absolute neutrophil count \<500/μL
•Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \>3 x the upper limit of normal (ULN); \>5 × ULN if associated with clinically suspected liver infiltration by mastocytosis or another disease for which the patient enrolled into the study
•Total bilirubin \>1.5 × ULN; \>3 × ULN if associated with liver infiltration by the disease being treated or in the presence of Gilbert's Disease (In the case of Gilbert's disease, a direct bilirubin \> 2.0 ULN would be an exclusion.)
•Estimated (Cockroft-Gault formula) or measured creatinine clearance \<40 mL/min
•Brain malignancy or metastases to the brain
•History of a seizure disorder or requirement for anti-seizure medication
•Known risk of intracranial bleeding, such as a brain aneurysm or history of subdural or subarachnoid bleeding
•Eosinophilia and known positivity for the FIP1L1-PGDFRA fusion, unless the patient has demonstrated relapse or progressive disease on prior imatinib therapy

Locations (11)

Stanford Cancer Institute

Stanford, California, United States

University of Colorado Cancer Center

Denver, Colorado, United States

Emory University

Atlanta, Georgia, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

University of Michigan Health System

Ann Arbor, Michigan, United States

Mount Sinai Hospital

New York, New York, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

MD Anderson Cancer Center

Houston, Texas, United States

Huntsman Cancer Institute

Salt Lake City, Utah, United States

Beatson West of Scotland Cancer Centre

Glasgow, United Kingdom

Key Dates

Start Date

March 10, 2016

Primary Completion Date

October 5, 2022

Completion Date

January 19, 2023

Last Updated

March 17, 2023

Enrollment

ACTUAL participants

Conditions

Aggressive Systemic MastocytosisSystemic Mastocytosis-associated Hematologic Non-mast Cell DiseaseMast Cell LeukemiaRelapsed or Refractory Myeloid Malignancies

Interventions

Avapritinib

DRUG

Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies

NCT01273766

Acute Undifferentiated LeukemiaAdult Acute Lymphoblastic Leukemia in Remission+134 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 17, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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