Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

Summary

This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).

Official Title

A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis

Keywords

Advanced Systemic Mastocytosis (AdvSM), SM With an Associated Hematologic Neoplasm (SM-AHN), Mast Cell Leukemia (MCL), Aggressive Systemic Mastocytosis (ASM), 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, Neoplasms, Mastocytosis, Systemic, Leukemia, Mast-Cell, Aggression, CGT9486 tablets

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Diagnosed with 1 of the following advanced mastocytosis diagnoses by Eligibility Committee
  2. Aggressive Systemic Mastocytosis (ASM)
  3. Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
  4. Mast Cell Leukemia (MCL)
  5. Measurable disease according to modified IWG-MRT-ECNM criteria. (A subset of patients inevaluble per mIWG-MRT-ECNM will be included in the study).
  6. ECOG (0 to 3)
  7. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits.

You CAN'T join if...

  1. Persistent toxicity from previous therapy for Advanced Systemic Mastocytosis that has not resolved to ≤ Grade 1
  2. Associated hematologic neoplasm requiring immediate antineoplastic therapy
  3. Clinically significant cardiac disease
  4. 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)
  5. Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
  6. History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
  7. Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment
  8. 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
  9. Received hematopoietic growth factor support within 14 days before the first dose of study drug
  10. . Received strong CYP3A4 inhibitors or inducers before the first dose of study drug
  11. . Need for treatment with steroids

Locations

  • UCLA Medical Center accepting new patients
    Los Angeles California 90095 United States
  • City of Hope Comprehensive Cancer Center accepting new patients
    Duarte California 91010 United States
  • Stanford Cancer Institute accepting new patients
    Stanford California 94305 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Cogent Biosciences, Inc.
ID
NCT04996875
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 140 study participants
Last Updated