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)
Persistent toxicity from previous therapy for AdvSM that has not resolved to ≤ Grade 1
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 demonstrate lack of PDGFRA fusion mutation prior to enrollment