A Study of CGT9486 in Patients with Advanced Systemic Mastocytosis
Research type
Research Study
Full 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
IRAS ID
299172
Contact name
Amanda Pilla
Contact email
Sponsor organisation
Cogent Biosciences, Inc.
Eudract number
2021-001010-10
Duration of Study in the UK
4 years, 1 months, 28 days
Research summary
The purpose of this study is to see how safe and effective the study drug, CGT9486, is as an inhibitor of the KIT gene in people that have advanced systemic mastocystosis (AdvSM). AdvSM is aggressive and life-threatening, disease progression is driven by the activation of the KIT receptor. Targeted therapies against KIT are emerging as potential treatments for AdvSM.
There are 2 main parts to this study. Participants cannot take part in both parts. Part 1 will determine the optimal dose for patients with AdvSM, each participant will be assigned to a dose group and will receive a specific dose of CGT9486 in tablet form, 50 mg :100 mg :200 mg twice daily: 400 mg once daily, for a minimum of two 28 day cycles. The dose that will be used in Part 2 of this study will be determined in Part 1.
Part 2 will consist of a dose expansion period, participants will receive the selected optimal dose based on the doses tested during Part 1 of the study.
The total number of planned patients with advanced systemic mastocytosis (AdvSM) enrolled in the study is approximately 140 and includes:
Part 1: Dose Optimisation will enroll approximately 60 participants
Part 2: Expansion will enroll a total of approximately 80 participantsREC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
21/EE/0217
Date of REC Opinion
27 Oct 2021
REC opinion
Further Information Favourable Opinion