A Study to Evaluate the Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Profiles of CGT9486 in subjects with Advanced Systemic Mastocytosis
- Conditions
- Advanced Systemic MastocytosisMedDRA version: 26.1Level: LLTClassification code: 10056453Term: Aggressive systemic mastocytosis Class: 10005329Therapeutic area: Diseases [C] - Neoplasms [C04]
- Registration Number
- CTIS2024-511407-42-00
- Lead Sponsor
- Cogent Biosciences Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 140
Diagnosed with 1 of the following advanced mastocytosis diagnoses: 1. Aggressive Systemic Mastocytosis (ASM) 2. Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN) 3. Mast Cell Leukemia (MCL), Measurable disease according to modified IWG-MRT-ECNM criteria, ECOG Status 0 to 3, Have clinically acceptable laboratory screening results (clinical chemistry, hematology) within certain limits, Other protocol-defined inclusion criteria apply.
Persistent toxicity from previous therapy for Advanced Systemic Mastocytosis that has not resolved to = Grade 1, Received strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives, whichever is longer, before the first dose of study drug., Need for treatment with high dose steroids, Associated hematologic neoplasm requiring immediate antineoplastic therapy, Clinically significant cardiac disease, Known positivity for the FIP1L1 PDGFRA fusion (patients with eosinophilia without detectable KIT D816V mutation must also lack the PDGFRA fusion mutation prior to enrolment), Seropositive for human immunodeficiency virus (HIV) 1 or 2, positive for hepatitis B surface antigen, or positive for hepatitis C virus (HCV) antibody, History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study, Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment, Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening bone marrow biopsy, Received hematopoietic growth factor support within 14 days before the first dose of study drug.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method