A Phase 2 Study of Bezuclastinib in Nonadvanced Systemic Mastocytosis
Research type
Research Study
Full 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
IRAS ID
1006436
Contact name
Kelvin Ho
Contact email
Sponsor organisation
Cogent Biosciences, Inc.
Eudract number
2022-000292-39
Clinicaltrials.gov Identifier
Research summary
This is a 3-part study of the bezuclastinib in subjects with Nonadvanced Systemic Mastocytosis (NonAdvSM), a rare mast cell disorder subtype characterized by accumulation and clonal proliferation of abnormal neoplastic mast cells in various organs.
There will be approximately 50 centers in North America, Europe, and other international locations
The total duration of the study is expected to be approximately 5 years. The total duration of study participation for individual subjects from Screening through the Extension Period is approximately 28 months and 31 months for Part 1 and Part 2 participants, respectively.
Screening Period (Part 1 and Part 2): Up to 42 days
Treatment Period (Part 1 and Part 2): From the first dose of study drug through completion of 3 cycles (12 weeks) in Part 1 or 6 cycles (24 weeks) in Part 2, or until intolerable toxicity, progression to advanced systemic mastocytosis (SM), or discontinuation due to subject withdrawal of consent or Investigator decision, whichever occurs first.
Extension Period (Part 3): Subjects who have completed the treatment period (Part 1 or Part 2) are eligible to receive treatment with bezuclastinib (CGT9486) for up to 2 years, or until intolerable toxicity, progression to advanced SM, or discontinuation due to subject withdrawal of consent or Investigator decision, whichever occurs first.
There are currently no approved therapies to treat the underlying disease of indolent systemic mastocytosis (ISM) or smoldering systemic mastocytosis (SSM) . Treatment is limited to trigger avoidance and use of anti-mediator drug therapy aimed at controlling symptoms of mast cell activation.
Bezuclastinib is an inhibitor of KIT with potent activity against KIT exon 17 and 18 activation-loop mutations. Preliminary clinical activity with bezuclastinib has been observed in a clinical study in subjects with gastrointestinal stromal tumors and advanced systemic mastocytosis.
REC name
Wales REC 1
REC reference
23/WA/0039
Date of REC Opinion
31 Aug 2023
REC opinion
Further Information Favourable Opinion