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Study of Mitapivat for Adults with Sickle Cell Disease

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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 sickle cell disease, a condition that affects the red blood cells, causing them to become misshapen and break down. The treatment being tested in this study is a medication called mitapivat, also known by its code name AG-348. The purpose of the study is to evaluate the safety and effectiveness of mitapivat in treating sickle cell disease.

Participants in the study will take mitapivat in the form of a tablet. The study will be conducted over a period of time, during which participants will receive the medication and be monitored for any changes in their condition. The study will assess how well the medication works in reducing the sickling of red blood cells, which is a key problem in sickle cell disease. Additionally, the study will look at the safety of the medication by observing any side effects or changes in health indicators such as vital signs and heart function.

Throughout the study, participants will undergo various assessments to track their health and response to the treatment. These assessments will include checking blood levels, evaluating the flexibility of red blood cells, and monitoring overall health and quality of life. The study aims to provide valuable information on how mitapivat can help manage sickle cell disease and improve the lives of those affected by this condition.

The research process

The trial runs in 6 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, the patient will begin the treatment phase. This involves taking the medication mitapivat, which is administered in the form of a tablet.

    The medication is taken orally, meaning it is swallowed. The specific dosage and frequency will be determined by the study protocol and communicated to the patient by the study team.

  2. Step 2

    Dose finding period

    During this period, the effectiveness of mitapivat is assessed by measuring changes in the patient's blood. This involves evaluating the 'Point of Sickling,' which is a measure of how the blood cells behave under certain conditions.

    The goal is to find the most effective dose that reduces sickling of the blood cells. This period helps determine the best dosage for the patient.

  3. Step 3

    Fixed dose extension period

    After the dose finding period, the patient will continue to take mitapivat at a fixed dose. This phase is designed to maintain the benefits observed during the dose finding period.

    The patient's response to the medication will be monitored regularly to ensure safety and effectiveness.

  4. Step 4

    Prolonged fixed dose extension period

    In this phase, the patient continues with the fixed dose of mitapivat for an extended period. The aim is to observe long-term effects and ensure sustained benefits.

    Regular check-ups and tests will be conducted to monitor the patient's health and any changes in their condition.

  5. Step 5

    Safety and efficacy evaluations

    Throughout the study, the safety of mitapivat is evaluated by monitoring any side effects or adverse reactions. This includes regular physical examinations and tests such as electrocardiograms (ECG).

    The effectiveness of the treatment is also assessed by measuring changes in the patient's condition, including blood tests and other health indicators.

  6. Step 6

    Completion of the study

    At the end of the study, the patient will undergo a final evaluation to assess the overall impact of the treatment.

    The study team will review all collected data to determine the success of the treatment and any long-term effects.

Who can join the trial?

12 criteria

  • Must be a male or female with a confirmed diagnosis of **sickle cell anemia** or **HbS/beta-thalassemia**. This can be confirmed through past lab tests or new tests during the screening.
  • Patients with a higher **albumin to creatinine ratio** are given priority, but both high and normal levels are eligible. This ratio is a measure used to check kidney function.
  • Women who can have children must have a negative pregnancy test during the screening period. Women of reproductive potential are those who have not had certain surgeries or have not naturally stopped having periods for at least 12 months.
  • Women who can have children and men who are fertile must agree to use two forms of birth control during the study and for a period after the last dose of the study drug.
  • Must have a history of **VOCs** (painful episodes due to blocked blood flow) and record of hospital stays for sickle cell complications in the past 24 months.
  • Must have experienced at least one VOC or hospital admission for sickle cell issues in the past 12 months, or have a history of certain sickle cell complications like bone damage, kidney problems, or eye issues.
  • Must be at least 16 years old. If 16 or 17, must be at a certain stage of physical development known as Tanner Stage 5.
  • Must have a **hemoglobin** level between 2.5 and 6.9 mmol/L during screening. Hemoglobin is a protein in red blood cells that carries oxygen.
  • If taking **hydroxyurea** (a medication for sickle cell disease), the dose must have been stable for at least 3 months before starting the study.
  • Must take or continue taking at least 0.7 mg of **folic acid** daily during the study. Folic acid is a type of vitamin B that helps make healthy red blood cells.
  • Must have adequate organ function, which includes specific levels of liver enzymes, bilirubin, creatinine, neutrophils, and platelets. These are all measures of how well your organs are working.
  • Must be willing and able to provide written consent and follow all study procedures for the duration of the study.

Who cannot join the trial?

5 criteria

  • Patients who do not have sickle cell disease cannot participate. Sickle cell disease is a condition where red blood cells, which carry oxygen, are shaped like a sickle or crescent, instead of being round.
  • Patients who are not within the specified age range for the study cannot participate. The study includes certain age groups, so if you are outside these groups, you cannot join.
  • Patients who are not part of the specified clinical trial groups cannot participate. This means the study is looking for people with specific characteristics or conditions.
  • Both male and female patients are eligible, so gender is not a reason for exclusion.
  • Patients who are considered part of a vulnerable population may not be able to participate. Vulnerable populations include groups that might need special protection or care.
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Investigated drugs

Mitapivat: This medication is being studied for its potential to help people with sickle cell disease. It works by affecting the way red blood cells function, which may help reduce the sickling of these cells. The trial aims to see how well mitapivat can improve the condition of patients by measuring changes in the blood's ability to carry oxygen. The study also looks at the safety of mitapivat, including any side effects or changes in health indicators like heart activity and physical health.

What is already known about the treatment

Mitapivat sulfate – This medication is administered orally and is currently being evaluated in clinical trials for its safety and effectiveness in treating sickle cell disease. It is not yet widely used in medical practice, as it is still under investigation. The main therapeutic indication for mitapivat sulfate is to improve the condition of patients with sickle cell disease by reducing the sickling of red blood cells. At the molecular level, mitapivat sulfate works by activating an enzyme called pyruvate kinase, which plays a role in the energy metabolism of red blood cells, helping them maintain their normal shape and function. It is classified pharmacologically as a pyruvate kinase activator.

Investigated diseases

Sickle Cell Disease – Sickle cell disease is a genetic disorder that affects the shape and function of red blood cells. Normally, red blood cells are round and flexible, allowing them to move easily through blood vessels. In sickle cell disease, some red blood cells become rigid and shaped like a crescent or sickle. These sickle-shaped cells can get stuck in small blood vessels, which can slow or block blood flow and oxygen to parts of the body. This can lead to episodes of pain, known as sickle cell crises, and can cause damage to organs over time. The disease is inherited and is most common in people of African, Mediterranean, or Middle Eastern descent.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515569-32-00Estimated enrolment10 patientsSponsorJulius Clinical Research B.V.

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).