(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Advanced Systemic Mastocytosis (AdvSM), SM With an Associated Hematologic Neoplasm (SM-AHN), Mast Cell Leukemia (MCL)
About this trial
This is an interventional treatment trial for Advanced Systemic Mastocytosis (AdvSM) focused on measuring Mastocytosis, Systemic Mastocytosis, Advanced Mastocytosis, Aggressive Mastocytosis, Hematologic Neoplasms, Mast Cell, Mast Cell Leukemia, Soft Tissue Neoplasms, Neoplasms by site, Skin Diseases, Immune Complex Diseases, Immune System Diseases, Hypersensitivity, Hematologic Diseases, Leukemia, Myeloid Leukemia, Acute Myeloid Leukemia, SM with Associated Hematologic Neoplasm, AdvSM, ASM, SM-AHN, MCL, Neoplasm, D816V, KIT D816V, AML, bezuclastinib, CGT9486, CGT, PLX, Connective Tissue Neoplasms, Urticaria Pigmentosa
Eligibility Criteria
Key Inclusion Criteria:
Diagnosed with 1 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.
Key Exclusion Criteria:
- Persistent toxicity from previous therapy for Advanced Systemic Mastocytosis 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 also lack the PDGFRA fusion mutation prior to enrollment)
- Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or 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
- Received strong CYP3A4 inhibitors or inducers before the first dose of study drug
- Need for treatment with steroids
Sites / Locations
- University of Alabama at Birmingham (UAB) HospitalRecruiting
- Mayo Clinic ArizonaRecruiting
- City of Hope Comprehensive Cancer CenterRecruiting
- UCLA Medical CenterRecruiting
- Stanford Cancer InstituteRecruiting
- Galiz Research
- Winship Cancer Institute - Emory UniversityRecruiting
- Rush University Medical CenterRecruiting
- Dana-Farber Cancer InstituteRecruiting
- Columbia University Irving Medical Center
- Cleveland Clinic Taussig Cancer CenterRecruiting
- MUSC Health University Medical CenterRecruiting
- The University of Texas MD Anderson Cancer CenterRecruiting
- Huntsman Cancer Institute - University of Utah HealthRecruiting
- Nepean HospitalRecruiting
- Gold Coast University HospitalRecruiting
- Peter MacCallum Cancer CentreRecruiting
- AKH Wien, UniversitatsklinikumRecruiting
- CHU de LiegeRecruiting
- University of Alberta HospitalRecruiting
- St. Michael's Hospital - Unity Health TorontoRecruiting
- Centre Hospitalier Universitaire (CHU) de PoitiersRecruiting
- Centre Hospitalier Universitaire (CHU) de ToulouseRecruiting
- University Hospital AachenRecruiting
- Universitätsklinikum FreiburgRecruiting
- IRCCS Azienda Ospedaliero Universitaria di BolognaRecruiting
- University Medical Center GroningenRecruiting
- Oslo University HospitalRecruiting
- Hospital Universitario Vall d'HebronRecruiting
- Institut Català d'Oncologia - Hospital Duran i ReynalsRecruiting
- Hospital Universitario Ramón y CajalRecruiting
- University College London Hospital - NHS Foundation TrustRecruiting
- Guy's Hospital - NHS Foundation TrustRecruiting
Arms of the Study
Arm 1
Experimental
bezuclastinib