AuthorisedTherapeutic confirmatory (Phase III)Interventional

The Effect of Mitapivat on Cerebral Perfusion and Oxygen Metabolism in Sickle Cell Disease

This study is investigating a drug called mitapivat for individuals living with sickle cell anaemia. The main goal is to understand how mitapivat influences blood flow and oxygen use in the brain. Researchers will use special MRI scans at 3 and 12 months to measure these changes. They're also looking at other health markers, including heart function, using different tests like echocardiograms. This research aims to see if mitapivat can help improve brain health and overall well-being for people with sickle cell anaemia, ultimately hoping to confirm if this treatment has a positive effect.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Amsterdam UMC Stichting

Enrolment target

Start

02 Jun 2025

Sickle cell anaemia

What is this study about?

This study is a Phase III clinical trial, which means it's one of the final steps in testing a new medication before it can be made widely available. It's designed for people who have sickle cell anaemia, a condition that affects red blood cells and can sometimes lead to complications in different parts of the body, including the brain.

The main purpose of this research is to understand how a new medicine called mitapivat affects the brain. Specifically, scientists want to see how much blood flows to the brain and how well the brain uses oxygen. These are important measures because problems with blood flow and oxygen can sometimes lead to issues for people with sickle cell anaemia. They will use special types of MRI scans, which are safe and painless, to check these things at two points in time: after 3 months and again after 12 months of treatment.

Beyond just the brain, the study will also look at other important health indicators. For example, they'll check heart function using a test called an echocardiogram, which uses sound waves to create images of your heart. This comprehensive approach helps researchers get a full picture of how mitapivat might affect different body systems. The overall goal is to gather strong evidence to confirm if mitapivat is an effective and safe treatment that can make a positive difference for people with sickle cell anaemia.

Key takeaways

This study is testing a new medicine called mitapivat for sickle cell anaemia.
It will carefully check how the medicine affects blood flow and oxygen use in the brain.
Special MRI scans and heart checks will be done at 3 and 12 months.
The study lasts for about 12 months.
It aims to find out if mitapivat can help improve health for people with sickle cell anaemia.
Participation is for adults aged 18 and over.

Who may be eligible?

This study is looking for volunteers who have sickle cell anaemia.

You must be an adult aged 18 years or older to take part. There is no upper age limit for joining this particular study.

Both men and women are welcome to participate in this research.

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.

Do you have sickle cell anaemia?
Are you 18 years old or older?
Are you able to attend appointments for up to 12 months?
Are you willing to have MRI scans and heart checks?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you would be taking a medicine called mitapivat. Throughout your participation, you would have several clinic visits over 12 months. During these visits, you will have special MRI scans to check blood flow and oxygen use in your brain. You will also have heart checks using an echocardiogram. These assessments will happen at 3 months and again at 12 months to see how you respond to the treatment over time. The total duration of your active involvement in the study would be about 12 months, with follow-up appointments scheduled during this period.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and risks. One potential benefit is that the study medicine, mitapivat, could improve your brain health and overall condition. However, it's also possible that you might not experience any direct benefit, or you could experience side effects from the medication or the test procedures. All known risks will be fully explained to you before you decide to participate. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands

Common questions

What is mitapivat?

Mitapivat is a medicine being studied to see if it can help people with sickle cell anaemia.

What is sickle cell anaemia?

Sickle cell anaemia is a blood disorder that affects red blood cells, which carry oxygen around the body.

What is an MRI scan?

An MRI scan is a safe, painless test that uses strong magnets and radio waves to create detailed pictures inside your body, in this case, your brain.

How long will I be in the study?

If you join, you will be in the study for about 12 months, with check-ups at 3 and 12 months.

Can I leave the study if I change my mind?

Yes, you can leave the study at any time, for any reason, and it will not affect your usual medical care.