UnknownDigestion, Kidneys & Other OrgansNIHR-supported projectCancer
An open-label, multi-centre, rollover study to characterise long-term safety and efficacy of Etavopivat in adults and children who have sickle cell disease or thalassemia and have completed a treatment period in an Etavopivat study
Recipient organisationNIHR Wellcome Trust Manchester Clinical Research Facility
NIHR supportRecorded as supported by this research centre
PeriodMar 2025 — Ongoing
In plain English
AI plain-English summary
People with sickle cell disease or thalassaemia who have already benefited from the experimental drug etavopivat will continue taking it for up to five more years to see if it remains safe and effective long-term. Sickle cell disease causes red blood cells to become stiff and sticky, blocking blood vessels and triggering painful crises that can damage organs. Thalassaemia destroys red blood cells, leading to chronic anaemia. Etavopivat activates a protein that helps red blood cells bind oxygen and produce energy, improving their health. Earlier short-term studies suggested it reduces symptoms, but no one knows whether those benefits hold up over years of daily use, or whether new side effects emerge. If the drug proves safe and effective long-term, it could become a chronic treatment that prevents pain crises and organ damage, letting people with these inherited blood disorders live with fewer hospital visits and less disability. The study tracks 325 participants aged 11 months and older through regular clinic visits, blood and urine tests, heart monitoring, and imaging scans. Researchers will also assess pubertal development in children, since long-term drug effects on growth and maturation are unknown.
View original technical description
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.
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