(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
Hematopoietic Malignancies
Leukemia
Unknown Primary
18 Years and older, Male and Female
CGT9486-21-202 (primary)
NCI-2022-01167
Summary
This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study
comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care
(BSC) with placebo plus BSC in patients with nonadvanced systemic mastocytosis
(NonAdvSM), including indolent systemic mastocytosis and smoldering systemic
mastocytosis, whose symptoms are not adequately controlled by BSC. This study will be
conducted in three parts. Patients in Parts 1a, 1b and 2 will receive bezuclastinib or
placebo, and may roll over onto Part 3 to receive treatment with bezuclastinib.
Eligibility
- Diagnosed with 1 of the following diagnoses according to the 2016 World Health Organization (WHO) classification for systemic mastocytosis (SM):
- Indolent systemic mastocytosis (ISM), including the bone marrow mastocytosis subvariant
- 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
Treatment Sites in Georgia
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