Actively Recruiting
Phase 2 Study of Bezuclastinib for Treating Advanced Systemic Mastocytosis and Related Conditions
Led by Cogent Biosciences, Inc. · Updated on 2026-07-31
140
Participants Needed
42
Research Sites
92 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating CGT9486, also known as bezuclastinib, in an open-label Phase 2 study for patients with Advanced Systemic Mastocytosis AdvSM. This includes those diagnosed with Aggressive Systemic Mastocytosis ASM, Systemic Mastocytosis with an Associated Hematologic Neoplasm SM-AHN, and Mast Cell Leukemia MCL. The study aims to evaluate the safety, effectiveness, pharmacokinetics, and pharmacodynamics of bezuclastinib in this patient population. Participants will receive bezuclastinib tablets orally, taken continuously in 28-day cycles. The study is divided into two parts Part I focuses on identifying effective and tolerable dosing exposures over 18 months, while Part II evaluates the drugs efficacy by measuring objective response rates and confirming the exposure-response relationship, also over 18 months. Additional assessments include effects on mutation allele burden, serum tryptase levels, histopathologic changes, spleen and liver volume, and safety monitoring. During the study, participants will undergo various clinical evaluations, including laboratory tests, imaging to monitor organ size changes, and assessments of disease response and progression. Researchers will track adverse events and pharmacokinetic profiles throughout the 18 months. The study involves continuous monitoring of participants to understand the treatments impact on survival and disease progression over this period.
CONDITIONS
Brief Title
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), or Mast Cell Leukemia (MCL) confirmed by Eligibility Committee
- Measurable disease according to modified IWG-MRT-ECNM criteria
- ECOG performance status between 0 and 3
- Clinically acceptable local laboratory screening results (clinical chemistry, hematology)
- Adults aged 18 years or older
You will not qualify if you...
- Persistent toxicity from previous therapy for AdvSM not resolved to Grade 1 or better
- Associated hematologic neoplasm requiring immediate antineoplastic therapy
- Clinically significant cardiac disease
- Positive for FIP1L1 PDGFRA fusion mutation
- Positive for HIV 1 or 2, hepatitis B surface antigen, or hepatitis C antibody
- History of significant bleeding within 30 days before first dose or need for therapeutic anticoagulation
- Malignancy other than AdvSM treated or diagnosed within prior 3 years
- Recent cytoreductive or investigational therapy within specified timeframes before screening
- Recent hematopoietic growth factor support within 14 days before first dose
- Use of strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives before first dose
- Need for treatment with high dose steroids
Research Team
C
Cogent Biosciences, Inc.
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