This study is in progress, not accepting new patients

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

a study on SSM Mastocytosis, Indolent Mastocytosis, Systemic Mastocytosis ISM BMM Smoldering Systemic Mastocytosis Bone Marrow Mastocytosis Hematologic Disease

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Los Angeles 5368361, California 5332921 and other locations
Dates
study started
study ends around

Description

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. Additionally, a substudy of subjects will investigate the efficacy, safety, and tolerability of bezuclastinib in patients who are experiencing inadequate symptom control with avapritinib.

Official Title

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

Keywords

SSM, Mastocytosis, Indolent, Mastocytosis, Systemic, Mastocytosis, ISM, BMM, Smoldering Systemic Mastocytosis, Bone Marrow Mastocytosis, 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, Bezuclastinib Tablets (Formulation A), Bezuclastinib Tablets (Formulation B), (Prior Therapy Sub-study) Bezuclastinib

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Diagnosed with 1 of the following diagnoses according to the 2016 World Health

    Organization (WHO) classification for systemic mastocytosis (SM):

  2. Moderate-to-severe symptoms based on a minimum total symptom scoew (TSS) of the Mastocytosis Activity Score (MAS) and after establishing a stable regimen of at least 2 antimediator therapies over a 14-day eligibility period
  3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0 to 2
  4. For patients receiving corticosteroids, the dose must be ≤10 mg/day of prednisone or equivalent

You CAN'T join if...

  1. Persistent toxicity from previous therapy for NonAdvSM that has not resolved to ≤ Grade 1
  2. 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
  3. Diagnosed with mastocytosis of the skin without systemic involvement
  4. Received prior treatment with any targeted KIT inhibitor with the exception of approved agents for the treatment of SM
  5. 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
  6. Received radiotherapy or psoralen and ultraviolet A therapy <14 days before starting screening assessments
  7. Received any hematopoietic growth factor support <14 days or 5 half lives of the drug before starting screening assessments
  8. History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
  9. Need for treatment of corticosteroids at >10 mg/day of prednisone or equivalent
  10. Received strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives before the first dose of study drug

Locations

  • University of California, Los Angeles (UCLA) - Medical Center
    Los Angeles 5368361 California 5332921 90095 United States
  • Modena Allergy and Asthma Clinical
    La Jolla 5363943 California 5332921 92037 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Cogent Biosciences, Inc.
ID
NCT05186753
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 237 study participants
Last Updated