Neoplasms

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Rare Diseases

Mastocytosis

Hypersensitivity

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Mastocytosis, Systemic

Immune Complex Diseases

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Mast Cell Activation Disorders

Systemic Mastocytosis

All Drugs and Chemicals

Antigen-Antibody Complex

Bezuclastinib will be administered orally, once daily continuously for 28-day cycles

Bezuclastinib Tablets (Formulation A)

Bezuclastinib Tablets (Formulation B)

Placebo will be administered orally, once daily continuously for 28-day cycles

Placebo Tablets

Hina Jolin, PharmD

Rachael Easton, MD, PhD

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.

In Part 1a and 1b of the study, patients with NonAdvSM will be randomly assigned to 1 of 2 dose levels of bezuclastinib plus BSC, or to placebo plus BSC. Upon analysis of the Part 1 data, a dose will be selected for Part 2. In Part 2, patients with NonAdvSM will be randomly assigned to the selected dose of bezuclastinib plus BSC, or to placebo plus BSC.

Patients who complete Part 1 or Part 2 may participate in Part 3 in which all patients will receive bezuclastinib plus BSC.

(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of The Safety and Efficacy of CGT9486 in Subjects With Nonadvanced Systemic Mastocytosis

Selection of the recommended dose to be used in subsequent parts of the study.

Mean absolute change in a disease-specific patient reported outcome (PRO)

Scale of 0-100, higher numbers represent more severe impairment to quality of life.

Scale of 0-100, higher numbers represent better symptom outcomes

Response rate based on reduction in disease specific PRO

Spots Global Cancer Trial Database for nonadvanced systemic mastocytosis