About this study
Sickle cell disease is a serious blood problem that changes how red blood cells work. Healthy red blood cells are round and soft. In sickle cell disease, the cells become hard, sticky, and shaped like a banana (sickle-shaped). They also break down more quickly than healthy cells. When they break down, they leave the body without enough red blood cells to carry oxygen, causing anemia.
Sickle cells can block blood flow and cause pain episodes called vaso-occlusive crises. They can also harm organs and make people feel very tired.
This study will test a new medicine called etavopivat. This medicine is designed to help red blood cells stay healthy and carry oxygen better. Researchers want to see if this medicine can lower the number of pain episodes and help people feel less tired. They want to see if it will help improve anemia, organ health, and the ability to exercise. They will also check if the medicine is safe to use.
People who join the study will be randomly assigned to get either etavopivat or a placebo (a pill that looks the same but has no medicine) for 1 year. After that, everyone will get etavopivat for another year. People in the study will take 2 tablets every day. They will visit the clinic about 20 times over 2 years. At these visits, doctors will check blood and urine (pee), do heart tests, and ask questions about pain and daily activities. Some visits will include a walking test to see how far the person can walk in 6 minutes.
The main goal is to find out if etavopivat is safe and if it works better than placebo to reduce pain crises. Researchers will also look at whether it improves blood health, reduces tiredness, and helps people feel better overall. Learning this could lead to new treatments that make life easier for people with sickle cell disease.
Eligibility overview
- Has sickle cell disease
- At least 12 years old
- Had 1–15 pain episodes in the past year