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

Phase 2

• Conditions: Advanced Mastocytosis; mast cell accumulation; myeloid neoplasm; 10047954

• Registration Number: NL-OMON53808
• Lead Sponsor: Cogent Biosciences, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

1. Diagnosed with 1 of the following advanced mastocytosis diagnoses by
Eligibility Committee
a. Aggressive Systemic Mastocytosis (ASM)
b. Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
c. Mast Cell Leukemia (MCL)

2. Measurable disease according to modified IWG-MRT-ECNM criteria. (A subset of
patients inevaluble per mIWG-MRT-ECNM will be included in the study).

3. ECOG (0 to 3)

4. Have clinically acceptable local laboratory screening results (clinical
chemistry, hematology) within certain limits.

Exclusion Criteria

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Part 1<br /><br>Safety assessments and dose modifications<br /><br>Pharmacokinetics (PK) and pharmacodynamic assessments<br /><br>Overall Response Rate (ORR)<br /><br><br /><br>Part 2 Stage 1<br /><br>• Safety assessments and dose modifications<br /><br>• PK and pharmacodynamic assessments<br /><br>• Overall Response Rate (ORR)<br /><br><br /><br>Part 2 Stage 2<br /><br>ORR</p><br>