(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent and Smoldering Systemic Mastocytosis



Status:Recruiting
Healthy:No
Age Range:18 - Any
Updated:3/21/2019
Start Date:March 2019
End Date:November 2023
Contact:Blueprint Medicines
Email:SM@blueprintmedicines.com
Phone:617-714-6707

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A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy

This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy
and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with
indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM) whose
symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All
patients will receive treatment with avapritinib during Part 3 including those rolling over
from the placebo group.


Key Inclusion Criteria:

- 1. Patient must have systemic mastocytosis (SM), confirmed by Central Pathology Review
of bone marrow biopsy, and indolent SM (ISM) or smoldering (SSM) subtype, as confirmed
by WHO diagnostic criteria. In Part 1 of the study, only patients with a diagnosis of
ISM are eligible.

- 2. Patient must have moderate-to-severe symptoms based on minimum mean total symptom
score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility
screening period.

- 3. Patient must have failed to achieve symptom control for 1 or more Baseline symptoms
measured by ISM-SAF.

- 4. For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or
equivalent, and the dose must be stable for ≥ 14 days.

- 5. Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status
(PS) of 0 to 2.

Key Exclusion Criteria:

- 1. Patient must not have received prior treatment with avapritinib.

- 2. Patient must not have had any cytoreductive therapy including but not limited to
masitinib and midostaurin, or investigational agent, < 14 days, and for cladribine,
interferon alpha, pegylated interferon, or antibody therapy < 28 days.

- 3. Patient must not have received radiotherapy or psoralen and ultraviolet A (PUVA)
therapy < 14 days.

- 4. Patient must not have received any hematopoietic growth factor < 14 days.

- 5. Patient must not have a QT interval corrected using Fridericia's formula (QTcF) of
> 450 msec.
We found this trial at
5
sites
450 Brookline Ave
Boston, Massachusetts 2215
617-632-3000
Principal Investigator: Daniel J. DeAngelo, MD, PhD
Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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75 Francis street
Boston, Massachusetts 02115
(617) 732-5500
Principal Investigator: Maria C. Castells, MD, PhD
Brigham and Women's Hosp Boston’s Brigham and Women’s Hospital (BWH) is an international leader in...
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Edegem,
Principal Investigator: Vito Sabato, PhD
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Edegem,
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New York, New York 10032
Principal Investigator: Mark L. Heaney, MD, PhD
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New York, NY
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Salt Lake City, Utah 84112
Principal Investigator: Michael Deininger, MD, PhD
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Salt Lake City, UT
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