Bezuclastinib for Advanced Systemic Mastocytosis

This study is testing a new oral medication called bezuclastinib for people with advanced systemic mastocytosis (AdvSM). This includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL). The study aims to find a safe and effective dose of bezuclastinib and see how well it works to shrink the disease. You may be able to join if you are 18 or older and have one of these advanced mastocytosis diagnoses with measurable disease.

Study design
This is an open-label, two-part Phase 2 study, meaning both you and your doctors will know you are receiving bezuclastinib. The study plans to enroll 140 participants.
What's involved
You would take bezuclastinib tablets continuously in 28-day cycles. The study will measure how well the treatment works over 18 months.
Compensation
Not stated in the trial record.
Follow-up
The main goals of the study are measured at 18 months after starting treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04996875

(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis

Recruiting
PHASE2Ages 18+InterventionalTreatment
Cogent Biosciences, Inc.
~140 participants
Updated 2026-07-31 on ClinicalTrials.gov
What's tested:bezuclastinib

At a glance

Recruiting sites
18 of 42 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part I: Identify clinically active and tolerable exposures of bezuclastinib in patients with AdvSM
Measured over 18 months
+1 more outcome measured
Advanced Systemic Mastocytosis (AdvSM)
SM With an Associated Hematologic Neoplasm (SM-AHN)
Mast Cell Leukemia (MCL)
Aggressive Systemic Mastocytosis (ASM)
42 sites across 29 states
Germany4
Italy4
California3
France3
Spain3
United Kingdom2
Alabama1
Arizona1
  • Rachael Easton, MD, Ph.D. · STUDY_DIRECTOR · Cogent Biosciences, Inc.

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  • Part I: Identify clinically active and tolerable exposures of bezuclastinib in patients with AdvSM18 months
  • Part II: - Determine efficacy of bezuclastinib as measured by mIWG Objective Response Rate (ORR) - Confirm the exposure-response relationship of bezuclastinib18 months