A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

This study is looking for children and teenagers with sickle cell disease. Participants must be between 6 months and 18 years old when they join the study. If you're under 18, your parent or guardian will need to give their permission for you to take part, and you'll also need to agree if you're old enough.

To be considered, you need to have a confirmed diagnosis of sickle cell disease, which can be shown by previous blood tests. Your haemoglobin level (a measure of red blood cells) also needs to be within a certain range. The study is specifically for those with more severe sickle cell disease, meaning you've had certain health issues related to your condition in the past year, such as several pain crises or hospital stays. Your doctor will be able to check all these details to see if you might be a good fit.

If you join this study, you'll be taking the study medicine, etavopivat, once a day by mouth for 96 weeks (that's about 1 year and 10 months). During this time, you'll have regular visits to the clinic so the study team can check on your health and how you're responding to the medicine. These visits will likely include physical exams, blood tests, and discussions about how you're feeling. After the 96-week treatment period, there will be one final check-up about a month later to make sure everything is okay.