Actively Recruiting
A Phase 2 Open-Label Study of Bezuclastinib for Advanced Systemic Mastocytosis Evaluating Safety, Efficacy, and Pharmacology
Led by Cogent Biosciences, Inc. · Updated on 2026-05-05
140
Participants Needed
42
Research Sites
92 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating CGT9486 bezuclastinib in a Phase 2 open-label study for adults with Advanced Systemic Mastocytosis AdvSM, including Aggressive Systemic Mastocytosis ASM, Systemic Mastocytosis with an Associated Hematologic Neoplasm SM-AHN, and Mast Cell Leukemia MCL. This study aims to evaluate the drugs safety, efficacy, pharmacokinetics, and pharmacodynamics in these conditions. Participants receive bezuclastinib tablets taken orally in continuous 28-day cycles. The study has two parts conducted over 18 months each Part I focuses on identifying active and tolerable doses, while Part II assesses treatment effectiveness by measuring the modified IWG Objective Response Rate ORR and confirms exposure-response relationships. During the study, participants will undergo evaluations including blood and bone marrow tests, imaging for spleen and liver size, and monitoring of mutation levels and serum tryptase. Researchers will track adverse events, duration and time to response, progression-free and overall survival. The total study duration includes these assessments over 18 months.
CONDITIONS
Brief Title
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- 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 by modified IWG-MRT-ECNM criteria or included subset
- ECOG performance status of 0 to 3
- Clinically acceptable local laboratory screening results in clinical chemistry and hematology
- For Substudy Population (Rollover or High-Risk Cohorts), additional specific diagnostic and therapy-related criteria apply
You will not qualify if you...
- Persistent toxicity from previous AdvSM therapy not resolved to Grade 1 or less
- Associated hematologic neoplasm requiring immediate antineoplastic therapy
- Clinically significant cardiac disease
- Positive for FIP1L1 PDGFRA fusion or related mutations if KIT D816V mutation not detected
- Positive serology for HIV 1 or 2, hepatitis B surface antigen, or hepatitis C antibody
- Significant bleeding event within 30 days before first study drug dose or need for therapeutic anticoagulation
- Recent diagnosis or treatment of malignancy other than AdvSM within past 3 years
- Recent use of cytoreductive therapy or investigational agents within 14 to 28 days before screening
- Recent hematopoietic growth factor support within 14 days before first study drug dose
- Recent use of strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives
- Need for treatment with high dose steroids
- Substudy exclusions include Philadelphia chromosome-positive malignancy, acute myeloid leukemia, allogenic stem cell transplant eligibility, contraindications to therapy, and prior investigational or cytoreductive therapy toxicity
Research Team
C
Cogent Biosciences, Inc.
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