(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
SSM, Mastocytosis, Indolent, Mastocytosis, Systemic
About this trial
This is an interventional treatment trial for SSM focused on measuring Systemic Mastocytosis, Immune Complex Diseases, Immune System Diseases, Hypersensitivity, Hematologic Diseases, NonAdvSM, D816V, KIT D816V, Bezuclastinib, CGT9486, CGT, PLX, Nonadvanced Systemic Mastocytosis, PLX9486, Indolent Systemic Mastocytosis, ISM, Smoldering Systemic Mastocytosis
Eligibility Criteria
Key Inclusion Criteria:
Diagnosed with 1 of the following diagnoses according to the 2016 World Health Organization (WHO) classification for systemic mastocytosis (SM):
- Indolent systemic mastocytosis (ISM)
- Smoldering systemic mastocytosis (SSM)
- Moderate-to-severe symptoms based on a disease-specific PRO and after establishing a stable regimen of at least 2 antimediator therapies over a 14-day eligibility period
- Eastern Cooperative Oncology Group (ECOG) Performance Status 0 to 2
- For patients receiving corticosteroids, the dose must be ≤10 mg/day of prednisone or equivalent
Key Exclusion Criteria:
- Diagnosed with any of the following WHO SM classifications: bone marrow mastocytosis, advanced systemic mastocytosis including SM with associated hematologic neoplasm, aggressive SM, mast cell leukemia; or mast cell sarcoma
- Diagnosed with mastocytosis of the skin without systemic involvement
- Received prior treatment with any targeted KIT inhibitor
- Received prior cytoreductive therapy or investigational agent for <14 days or 5 half- lives of the drug and for cladribine, interferon alpha, pegylated interferon, or antibody therapy <28 days or 5 half-lives of the drug (whichever is longer), before starting screening assessments
- Received radiotherapy or psoralen and ultraviolet A therapy <14 days before starting screening assessments
- Received any hematopoietic growth factor support <14 days before starting screening assessments
- History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
- Need for treatment of corticosteroids at >10 mg/day of prednisone or equivalent
Sites / Locations
- Mayo Clinic ArizonaRecruiting
- One of a Kind Clinical Research CenterRecruiting
- Modena Allergy and Asthma ClinicalRecruiting
- Innovative Research of West FloridaRecruiting
- Maya Research CenterRecruiting
- Mayo Clinic - JacksonvilleRecruiting
- Mid Florida Hematology and Oncology CenterRecruiting
- Emory UniversityRecruiting
- Rush UniversityRecruiting
- Walter Reed National Military Medical CenterRecruiting
- Allervie Clinical ResearchRecruiting
- Chesapeake Research CenterRecruiting
- Brigham and Women's HospitalRecruiting
- Dana Farber Cancer InstituteRecruiting
- University of MichiganRecruiting
- Mayo Clinic- RochesterRecruiting
- Washington University at St. LouisRecruiting
- Dartmouth Hitchcock Medical CenterRecruiting
- Duke UniversityRecruiting
- The Ohio State UniversityRecruiting
- Vanderbilt University Medical CenterRecruiting
- AIR CareRecruiting
- The University of Texas MD Anderson Cancer CenterRecruiting
- Royal Prince Alfred HospitalRecruiting
- Royal Adelaide HospitalRecruiting
- Toronto Allergy and Asthma ClinicRecruiting
- AP-HP- Hopital Pitie-SalpetriereRecruiting
- CHU de Toulouse - Hopital LarreyRecruiting
- Universitaetsklinikum Aachen, AoeRRecruiting
- Charité Universitätsmedizin BerlinRecruiting
- University Medical Centre MannheimRecruiting
- Fondazione IRCCS Policlinico San MatteoRecruiting
- Policlinico Universitario Agostino GemelliRecruiting
- University Medical Center GroningenRecruiting
- Hospital Universitario Vall d'HebronRecruiting
- Hospital Universitario Ramon y CajalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm 8
Arm 9
Experimental
Experimental
Placebo Comparator
Experimental
Experimental
Placebo Comparator
Experimental
Placebo Comparator
Experimental
(Part 1a) Bezuclastinib Dose 1 + BSC
(Part 1a) Bezuclastinib Dose 2 + BSC
(Part 1a) Placebo + BSC
(Part 1b) Bezuclastinib Dose 1 + BSC
(Part 1b) Bezuclastinib Dose 2 + BSC
(Part 1b) Placebo + BSC
(Part 2) Bezuclastinib Selected Dose + BSC
(Part 2) Placebo + BSC
(Part 3) Bezuclastinib + BSC