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Study on the Effects of Mitapivat on Brain Blood Flow and Oxygen Use in Patients with Sickle Cell Anemia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Mitapivat on individuals with Sickle Cell Disease. Sickle Cell Disease is a genetic condition that affects the shape and function of red blood cells, leading to various health complications. The medication being tested, Mitapivat, is taken in the form of a tablet and is designed to help improve the way oxygen is used in the brain.

The purpose of the study is to understand how Mitapivat affects brain oxygen use and blood flow in people with Sickle Cell Disease. Participants in the study will take Mitapivat and undergo several tests, including MRI scans, over a period of 12 months. These tests will help researchers see how the medication impacts brain function and blood circulation.

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the medication. The study aims to provide valuable information on whether Mitapivat can be an effective treatment for improving brain oxygen use in individuals with Sickle Cell Disease.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participants will have their eligibility confirmed. This includes verifying the sickle cell disease genotype and ensuring hemoglobin levels are 10.5 g/dL or lower.

    Participants will provide documented informed consent, which involves reviewing and signing a consent form.

  2. Step 2

    Medication administration

    Participants will begin taking the medication mitapivat in tablet form. The medication is taken orally.

    The dosage, frequency, and duration of administration will be specified by the study team and should be followed as instructed.

  3. Step 3

    First assessment

    After three months, participants will undergo an MRI scan to assess the effect of mitapivat on cerebral oxygen metabolism.

    Additional assessments will include measuring cerebral blood flow and other cardiovascular parameters.

  4. Step 4

    Second assessment

    After twelve months, participants will have a second MRI scan to further evaluate the effects of mitapivat on cerebral oxygen metabolism.

    The same assessments conducted at three months will be repeated to monitor changes over time.

  5. Step 5

    Completion of study

    Upon completion of the study, participants will have a final review with the study team to discuss the results and any next steps.

    Participants will be informed about the findings related to their participation in the study.

Who can join the trial?

6 criteria

  • Must have a documented diagnosis of Sickle Cell Disease (SCD), specifically the genotypes HbSS or HbSβ0-thalassemia. This can be based on past lab tests or confirmed during the screening process.
  • Must be 18 years old or older.
  • Must have a hemoglobin level of 10.5 grams per deciliter (g/dL) or less. Hemoglobin is a protein in red blood cells that carries oxygen.
  • If taking hydroxyurea (HU), a medication used to treat SCD, the dose must be stable for at least 90 days before joining the study, with no expected changes needed.
  • Female participants who can have children must agree to either not have sexual intercourse as part of their usual lifestyle or use a highly effective method of birth control. Since the study drug, mitapivat, might reduce the effectiveness of hormonal birth control, women using these methods must also use an additional barrier method, like condoms, while in the study and for at least 28 days after the last dose. Women using non-hormonal birth control methods do not need an extra barrier method.
  • Must have provided informed consent, meaning they have agreed to participate after understanding the study details. This includes signing a consent form, and if applicable, the participant's legal representative or guardian must also agree.

Who cannot join the trial?

1 criterion

  • Patients with **sickle cell anaemia** cannot participate. This is a condition where red blood cells, which carry oxygen around the body, are shaped like a sickle or crescent, instead of being round.
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Verified sites

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  • Verified

    Amsterdam UMC

    The NetherlandsAmsterdam, The Netherlands

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Investigated drugs

Mitapivat is a medication being studied for its potential effects on sickle cell disease. It works by activating an enzyme in red blood cells, which can help them function better. In this trial, researchers are looking at how mitapivat might improve blood flow and oxygen use in the brain for people with sickle cell disease. This could potentially help reduce some of the complications associated with the disease, such as pain and organ damage. The goal is to see if mitapivat can make a positive difference in the way the brain receives and uses oxygen in patients with this condition.

What is already known about the treatment

Mitapivat – Mitapivat is administered orally in the form of tablets. It is currently being studied in clinical trials for its potential benefits in treating sickle cell anemia, a condition where red blood cells become misshapen and can block blood flow. The main therapeutic indication for mitapivat is to improve cerebral blood flow and oxygen metabolism in patients with sickle cell disease. At the molecular level, mitapivat works by activating an enzyme called pyruvate kinase, which plays a crucial role in the energy production of red blood cells, helping them function more effectively. It is classified pharmacologically as a pyruvate kinase activator.

Investigated diseases

Sickle Cell Anemia – Sickle cell anemia is a genetic blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This causes red blood cells to become rigid, sticky, and shaped like sickles or crescent moons. These misshapen cells can block blood flow, leading to pain and potential organ damage. Over time, the disease can cause anemia, as the sickle cells break down prematurely. The condition can also lead to episodes of pain, known as sickle cell crises, which occur when blood flow is obstructed. Chronic complications may include damage to organs such as the spleen, liver, and kidneys.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513528-41-02Estimated enrolment20 patientsSponsorAmsterdam UMC Stichting

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).