Safety and efficacy study of Etavopivat in adolescent patients with SCD
Research type
Research Study
Full title
A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients with Sickle Cell Disease
IRAS ID
1005669
Contact name
Nadia Success
Contact email
Sponsor organisation
Forma Therapeutics, Inc.
Eudract number
2022-001689-36
Research summary
Etavopivat (FT-4202) is being developed for and is intended for use as a treatment for patients with sickle cell disease (SCD) and other inherited blood disorders and diseases that primarily affect red blood cells. SCD is a blood disorder caused by the abnormal haemoglobin that damages and deforms red blood cells.
This study is being done to learn about etavopivat, a once a day medicine taken by mouth – in adolescents with sickle cell disease. Etavopivat activates an enzyme in red blood cells that may improve sickle cell complications.
The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Etavopivat has been studied in adults and a few adolescents in a small, early phase study and was well-tolerated and showed potential for benefit in sickle cell disease (SCD).
This is a Phase 1/2 study and up to 50 participants with SCD, aged 12 to < 18 years, are expected to enter the study.
The duration of study for an individual patient may last up to 105 weeks and includes the screening period (up to 5 weeks before study treatment), the 24-week primary treatment period, the 72-week extension treatment period, and an End of Study visit at 4 weeks after last full dose of study drug.
Additional treatment beyond 96 weeks may be offered on a separate post-trial expanded access study.
The study is sponsored by Forma Therapeutics, Inc.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
22/NE/0161
Date of REC Opinion
4 Nov 2022
REC opinion
Further Information Favourable Opinion