(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
- Conditions
- Advanced Systemic Mastocytosis (AdvSM)SM With an Associated Hematologic Neoplasm (SM-AHN)Mast Cell Leukemia (MCL)Aggressive Systemic Mastocytosis (ASM)
- Interventions
- Registration Number
- NCT04996875
- Lead Sponsor
- Cogent Biosciences, Inc.
- Brief Summary
This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 140
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description bezuclastinib bezuclastinib -
- Primary Outcome Measures
Name Time Method Part II: - Determine efficacy of bezuclastinib as measured by mIWG Objective Response Rate (ORR) - Confirm the exposure-response relationship of bezuclastinib 18 months Part I: Identify clinically active and tolerable exposures of bezuclastinib in patients with AdvSM 18 months
- Secondary Outcome Measures
Name Time Method Pure Pathologic Response (PPR) 18 months Months
Safety of CGT9486 as assessed by incidence of Adverse Events (AEs) 18 months Incidence of AEs according to CTCAE version 5.0 or higher
To determine the effects of bezuclastinib on mutation allele burden. 18 months Percentage change in KIT D816V
To determine the effects of bezuclastinib on serum tryptase. 18 months Percentage change in Serum Tryptase
To assess the pharmacokinetics of bezuclastinib in subjects with AdvSM. 18 months Percentage change in plasma concentrations of bezuclastinib
Change from baseline in histopathologic findings in blood and bone marrow 18 months Percentage change in mast cell infiltration in the bone marrow and percentage change in eosinophilia and monocytosis in the blood
Change in spleen and liver volume by imaging 18 months Percentage change
Duration of Response (DOR) 18 months Months
Time to Response (TTR) 18 months Months
Progression Free Survival (PFS) 18 Months Months
Overall Survival (OS) 18 months Months
Related Research Topics
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Trial Locations
- Locations (42)
University of Alabama at Birmingham (UAB) Hospital
🇺🇸Birmingham, Alabama, United States
Mayo Clinic Arizona
🇺🇸Phoenix, Arizona, United States
City of Hope Comprehensive Cancer Center
🇺🇸Duarte, California, United States
UCLA Medical Center
🇺🇸Los Angeles, California, United States
Stanford Cancer Institute
🇺🇸Stanford, California, United States
Galiz Research
🇺🇸Hialeah, Florida, United States
Winship Cancer Institute - Emory University
🇺🇸Atlanta, Georgia, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
Scroll for more (32 remaining)University of Alabama at Birmingham (UAB) Hospital🇺🇸Birmingham, Alabama, United States