FORMA 4204-HEM-301
Research type
Research Study
Full title
An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease
IRAS ID
289626
Contact name
Kate Gardner
Contact email
Sponsor organisation
Syneos Health UK
Eudract number
2020-003884-25
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
141464, IND #
Duration of Study in the UK
2 years, 2 months, 0 days
Research summary
This clinical trial is a Phase 2/3 study that will evaluate the effectiveness and safety of FT-4202 (the study drug being investigated) and test how well it works compared to placebo (a pill that looks like the study drug but it does not contain any medicine). The study is made up of 2 parts: Double-blind (DB) treatment period, where subjects will take the study drug or placebo, depending on which treatment group they are in, for 52 weeks; and the Open Label Extension period, where subjects who complete the double-blind period maybe eligible to receive the study drug in a 52-week open label extension.
Initially, patients will be randomized 1:1:1 to one of two dose levels of FT-4202 or placebo. There are two planned interim analyses in this study, one to select one of two FT-4202 dose levels and another to test the 24-week Hb response rate and the annualized VOC rate for futility, and the 24-week Hb response rate for efficacy. The effect of the drug will be measured against the placebo using the Hb response rate at Week 24 during the blinded treatment period and the annualized VOC rate during the 52-week blinded treatment period based on adjudicated VOC review. This study is sponsored by Forma Therapeutics, Inc. and is a global multi-center study taking place in the US, UK, Canada, France, Spain, Germany and Italy with the plan to enroll approximately 344 patients, male & female, aged 12 to 65 years.REC name
Wales REC 5
REC reference
21/WA/0165
Date of REC Opinion
5 Jul 2021
REC opinion
Further Information Favourable Opinion