A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease
Sickle cell disease is an inherited condition that causes red blood cells to become unusually shaped and break down quickly. This can lead to severe anaemia, meaning many people need regular blood transfusions to stay well. This Phase 3 study looks at an experimental medicine called mitapivat. Researchers want to see if mitapivat can help red blood cells survive longer, reducing or completely removing the need for blood transfusions over a 52-week period. Participants will be placed into one of two groups by chance: one group takes mitapivat tablets, and the other takes matching dummy tablets (placebo). Neither the participants nor their doctors will know who is getting which tablet. By comparing the two groups over a year, doctors hope to learn whether mitapivat could become an effective new treatment option for people living with sickle cell disease.
At a glance
What is this study about?
Sickle cell disease affects red blood cells, which carry oxygen around your body. In people with this condition, the cells can become hard, sticky, and shaped like a crescent or sickle. These abnormal cells do not live as long as healthy cells, often leading to severe tiredness, painful episodes, and anaemia. Because of this, many people need regular blood transfusions to replace damaged cells with healthy donor blood.
While blood transfusions are life-saving, having them frequently can be challenging. Over time, frequent transfusions can cause complications, such as too much iron building up in the body. Researchers are looking for treatments that help the body protect its own red blood cells so that transfusions are needed much less often.
This clinical trial is testing a medicine called mitapivat. Mitapivat is designed to boost a natural enzyme inside red blood cells, giving them more energy so they can stay healthy and survive longer. In this Phase 3 trial, doctors are comparing mitapivat against a placebo (a dummy pill) to see whether it safely helps adults with sickle cell disease go for a full year without needing a transfusion.
Key takeaways
- The trial tests whether mitapivat can safely stop or reduce the need for blood transfusions.
- Open to adults aged 18 and over who have sickle cell disease.
- Participants take either mitapivat or a dummy tablet (placebo) for 52 weeks.
- Regular hospital appointments and blood tests are required to monitor health.
- Joining is completely voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with sickle cell disease and currently need blood transfusions as part of their care. Both men and women can take part.
To join, participants must be well enough to attend regular hospital check-ups and be willing to take study tablets every day for at least 52 weeks.
You may not be eligible if you have certain other serious medical conditions, such as severe liver or kidney problems, or if you are pregnant or breastfeeding. The study team will review your complete medical history and blood test results to make sure it is safe for you to take part.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am 18 years of age or older.
- I have a confirmed diagnosis of sickle cell disease.
- I currently receive blood transfusions for my condition.
- I am able to attend regular hospital appointments for at least one year.
- I am not currently pregnant or planning a pregnancy during the study.
What does participation involve?
Taking part in this study lasts for at least 52 weeks (one year). If you join, you will be assigned by random chance to take either mitapivat or a matching placebo tablet every day by mouth. Neither you nor your doctor can choose your group. Throughout the year, you will visit the hospital clinic regularly for routine check-ups. These visits will involve blood tests to measure your red blood cell levels, physical examinations, and chats about how you are feeling. Doctors will carefully track whether you need any blood transfusions during the year. If you ever need a transfusion for your health, you will always receive one.
Potential risks and benefits
Locations (4)
- —UnverifiedItaly
- —UnverifiedFrance
- —UnverifiedBelgium
- —UnverifiedIreland
Common questions
What is a placebo and why is it used?
A placebo is a dummy tablet that looks identical to the real medicine but contains no active ingredients. It is used so researchers can clearly tell if health changes are genuinely caused by mitapivat.
Will I still get a blood transfusion if I become unwell?
Yes. Your safety and health always come first. If your doctor determines that you need a blood transfusion at any point during the study, you will receive one immediately.
How is the trial medication taken?
The study medicine is taken as an oblong, blue tablet by mouth once or twice daily, as directed by the study team.
Does taking part cost anything?
No. The study medication and all trial-related appointments and tests are provided free of charge, and travel expenses are often reimbursed.
Can I leave the study if I change my mind?
Yes. You have the right to withdraw from the trial at any time, for any reason, without it affecting your standard medical treatment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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