7822 FLORAL: Long-term treatment with etavopivat in people with sickle cell disease or thalassaemia
Research type
Research Study
Full title
An open-label, multi-centre, rollover study to characterise long-term safety and efficacy of Etavopivat in adults, adolescents and children who have sickle cell disease or thalassaemia and have completed a treatment period in an Etavopivat study
IRAS ID
1010509
Contact name
Clinical Transparency (2834)
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2024-510805-27
ISRCTN Number
Not applicable
Clinicaltrials.gov Identifier
Not applicable
Research summary
People with sickle cell disease produce unusually shaped red blood cells (sickle cells). These are stiff and sticky and can cause blood vessels to get blocked causing pain, referred to as vaso-occlusive crises (sickle cell pain crises). This can also damage vital organs and tissue.
People with thalassaemia do not make enough haemoglobin or make abnormal haemoglobin, which can cause red blood cells to be destroyed, and cause anaemia.
Etavopivat is a small molecule that activates a specific protein found in red blood cells. This protein helps red blood cells to bind oxygen and to produce energy, which improves red blood cell health, and may help reduce the symptoms of sickle cell disease and thalassaemia.
This study will help us understand how safe etavopivat is when taken for a longer time, and how well it works in people with sickle cell disease or thalassaemia.
This study may last for up to 264 weeks, or until etavopivat is approved for marketing in the participating countries.
In this study there will be:
A transfer visit, the same day as the last visit in the participants’ previous etavopivat study so participants can continue their etavopivat treatment without a gap.
A treatment period of up to 260 weeks. Participants will take etavopivat once every day and visit the clinic every 12 weeks for health checks.
A follow-up visit 4 weeks after the last dose of etavopivat, to check the participants’ health.
Approximately 325 participants who are 11 months old or older and have sickle cell disease or thalassaemia will take part. All participants will have been in an etavopivat study before joining and have benefitted from taking etavopivat.
Participants will have up to 24 clinic visits to collect information about their symptoms and perform different tests, including blood and urine samples, to monitor heart health and body temperature. Some participants will have imaging scans. There will also be a check to find out how far children are in puberty.REC name
London - Westminster Research Ethics Committee
REC reference
24/LO/0672
Date of REC Opinion
31 Oct 2024
REC opinion
Further Information Favourable Opinion