7807 Hibiscus2- A research study to evaluate how well Etavopivat works in people with sickle cell
Research type
Research Study
Full title
A global phase 3, randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease
IRAS ID
1010273
Contact name
Clinical Transparency (2834)
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2023-509175-16
Clinicaltrials.gov Identifier
Research summary
All organs in our body need oxygen to function optimally. Red blood cells are responsible for carrying oxygen from the lungs to all parts of the body. People with sickle cell disease produce unusually shaped red blood cells (sickle cells), that are stiff and sticky. This can cause blood vessels to get blocked causing pain, usually referred to as vaso-occlusive crises. This blockage of blood flow can also damage vital organs and tissue. Etavopivat is a small molecule that can activate a specific protein found in the red blood cells. This enzyme helps the red blood cells to produce energy and to bind oxygen, improving the health of red blood cells.
We are doing this study to see how well etavopivat, the new medicine being tested, works in people living with sickle cell disease. We will evaluate if etavopivat reduces the patients' number of vaso-occlusive crisis and damage to organs. We will also evaluate whether etavopivat improves the patients' exercise capacity and reduces their fatigue.
In the first 52 weeks participants will take etavopivat or placebo daily assigned by chance. In the second 52 weeks participants will take etavopivat daily.
A total of 408 male and female adolescent and adults, older than 12 years with sickle cell disease across the world will participate in this study. To be able to be part of the study, participants should have between 1 and 15 vaso-occlusive crises (sickle cell pain crises) in the past year and have low red blood cell count (anaemia).
The participants will be asked to come to 20 visits at the clinic. The participants will have physical examinations as well as other tests, including a walking exercise, heart checks and blood and urine tests. During specific clinic visits, participants will be asked to answer short questionnaires about their health and quality of life. Every day during the study, participants will use the Study App to register when they took the study medicine and register if they had any sickle cell pain.REC name
London - Riverside Research Ethics Committee
REC reference
24/LO/0467
Date of REC Opinion
30 Aug 2024
REC opinion
Further Information Favourable Opinion