A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis
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).
• Diagnosed with one of the following advanced mastocytosis diagnoses by Eligibility Committee
‣ Aggressive Systemic Mastocytosis (ASM)
⁃ Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
⁃ Mast Cell Leukemia (MCL)
• Measurable disease according to modified IWG-MRT-ECNM criteria. (A subset of patients inevaluble per mIWG-MRT-ECNM will be included in the study).
• ECOG (0 to 3)
• Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits
∙ Rollover Cohort
• Demonstrate AHN progression requiring immediate AHN-directed therapy while receiving bezuclastinib
• Demonstrated clinical benefit from bezuclastinib therapy
• Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits
∙ High-Risk Cohort
• Receiving or indicated for AHN-directed therapy.
• Diagnosed with one of the following pathologic diagnoses of SM-AHN:
‣ Myelodysplastic syndrome (MDS) that is high- or very high-risk
⁃ Accelerated phase myeloproliferative neoplasm (MPN)
⁃ MDS with excessive blasts in bone marrow or peripheral blood
⁃ Chronic myelomonocytic leukemia-2 (CMML-2)
• Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits.