A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With SCD
Research type
Research Study
Full title
A Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose-Finding, Efficacy, and Safety Study of Tebapivat in Participants With Sickle Cell Disease
IRAS ID
1011853
Contact name
William Cook
Contact email
Sponsor organisation
Agios Pharmaceuticals Inc.
Research summary
Sickle cell disease (SCD) is a blood disorder passed down from parents to children. It affects a protein found in red blood cells (RBCs) called haemoglobin. The abnormal haemoglobin causes RBCs to change shape. These sickled RBCs can clump together and block blood flow around the body. When blood vessels become blocked, this can cause painful episodes called sickle cell pain crises (SCPCs). Over time, the damage caused by pain crises and haemolytic anemia can lead to serious problems such as kidney, heart, and liver disease.
The study drug (called Tebapivat) attaches to a specific protein that may give red blood cells more energy and help more haemoglobin molecules bind to oxygen. These changes may reduce the opportunity for haemoglobin to stick together and form sickled cells, helping the body to improve red blood cell levels.
The purpose of this study is to look at the efficacy (how well this drug works to treat and prevent complications) and safety of tebapivat when administered with standard treatment for SCD.
About 56 participants worldwide will be enrolled into the study.
The study will be run in two periods. In double- blind period, the participants will receive either tebapivat or placebo for 12 weeks and after completion, the participants can join the open-label extension period to receive tebapivat for up to 52 weeks.
There will be several assessments (such as medical history, physical exams, vital signs, ECHO, ECGs, blood/urine samples, health questionnaires) to be completed at visits.REC name
London - Brent Research Ethics Committee
REC reference
25/LO/0343
Date of REC Opinion
29 May 2025
REC opinion
Further Information Favourable Opinion