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Study on the Safety of Mitapivat for Adults with Red Blood Cell Membrane Disorders

Verified siteRegistered drugNo placebo
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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 safety and effectiveness of a medication called mitapivat in adults with a condition known as erythrocyte membranopathies. Erythrocyte membranopathies are disorders that affect the membranes of red blood cells, which can lead to various health issues. The medication being tested, mitapivat, is taken in the form of film-coated tablets and is also known by its code name, AG-348. The purpose of this study is to evaluate how safe mitapivat is for patients with this condition.

Participants in the study will receive different doses of mitapivat over a period of time, with the maximum treatment period being 56 days. The study will involve regular check-ups and monitoring to assess the safety of the medication. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects. The study aims to gather information on any side effects or changes in health that occur while taking mitapivat.

Throughout the study, participants will undergo various assessments, including blood tests and physical examinations, to monitor their health and the impact of the medication. The study will also look at changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen, as well as other health indicators. The goal is to determine if mitapivat is a safe and effective treatment option for people with erythrocyte membranopathies.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a physical examination.

    Blood tests are performed to measure hemoglobin levels and other relevant markers. These tests help determine the baseline health status.

  2. Step 2

    Screening period

    During the screening period, which lasts up to 50 days, additional tests are conducted to ensure all eligibility criteria are met.

    Participants must have a hemoglobin concentration below specified levels and meet other health criteria.

  3. Step 3

    Medication administration

    Participants begin taking mitapivat, available in 5 mg, 20 mg, and 50 mg film-coated tablets, orally.

    The dosage and frequency are determined based on individual needs and study requirements.

  4. Step 4

    Fixed dose period 1

    Participants continue taking the assigned dose of mitapivat for a specified period.

    Regular monitoring of hemoglobin levels and other health indicators is conducted to assess the response to the medication.

  5. Step 5

    Fixed dose period 2

    The dosage may be adjusted based on the results from the first fixed dose period.

    Continued monitoring of health indicators and any side effects is performed to ensure safety and efficacy.

  6. Step 6

    Safety and efficacy evaluation

    Throughout the study, the safety of mitapivat is evaluated by monitoring for any adverse events.

    Regular assessments include clinical laboratory tests, ECGs, physical examinations, and DEXA scans.

  7. Step 7

    Study completion

    Upon completion of the study, a final assessment is conducted to evaluate the overall health status.

    Participants may receive follow-up care as needed based on the study findings.

Who can join the trial?

12 criteria

  • Must be a male or female with a condition called RBC membranopathy or congenital dyserythropoietic anemia type II (CDAII). The diagnosis should be confirmed by a genetic test.
  • Must be 18 years or older at the time of the first screening.
  • Must have an average hemoglobin (Hb) concentration below 13.0 g/dL for males and 11.0 g/dL for females. If the average Hb is above 10.0 g/dL, the patient must also have one of the following:
    • Splenomegaly: An enlarged spleen measuring 12.5 cm or more.
    • Fatigue due to the breakdown of red blood cells.
    • Active breakdown of red blood cells, shown by tests like haptoglobin, bilirubin, LDH, or reticulocytes.
    • Must take at least 0.8 mg of folic acid daily during the study.
    • Must have adequate organ function, which includes:
      • Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels should be no more than 2.5 times the upper limit of normal, unless the increase is due to red blood cell breakdown or iron in the liver.
      • Normal or elevated serum bilirubin levels. If elevated, it should be due to red blood cell breakdown and not related to other liver or bile duct issues.
      • Estimated glomerular filtration rate should be 45 mL/min/1.73 m² or higher, which indicates kidney function.
      • Must be willing to give written consent and follow all study procedures.
      • Women who can have children must have a negative pregnancy test before the study starts.
      • Women who can have children and men with partners who can have children must either not have sex or agree to use two forms of birth control, one of which must be highly effective, during the study and for a period after the study ends.

Who cannot join the trial?

1 criterion

  • Individuals with erythrocyte membranopathies cannot participate. This refers to conditions affecting the outer layer of red blood cells, which can cause them to function improperly.
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Investigated drugs

Mitapivat is a medication being studied for its safety and effectiveness in adult patients with erythrocyte membranopathies. It is designed to help improve the function of red blood cells in people with certain blood disorders. The trial aims to assess how well patients tolerate the medication and its potential benefits in managing their condition.

What is already known about the treatment

Mitapivat – Mitapivat is administered orally in tablet form. It is currently being studied in clinical trials for its safety and effectiveness in treating erythrocyte membranopathies, which are disorders affecting the red blood cell membrane. The main therapeutic indication for mitapivat is to improve the function of red blood cells in these conditions. 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. It is classified pharmacologically as a pyruvate kinase activator.

Investigated diseases

Erythrocyte Membranopathies – Erythrocyte membranopathies are a group of disorders affecting the membrane of red blood cells, leading to their abnormal shape and function. These conditions often result in the premature destruction of red blood cells, causing anemia. The red blood cells may become more fragile and less flexible, which can lead to their breakdown as they pass through the circulatory system. Symptoms can include fatigue, jaundice, and an enlarged spleen due to the increased breakdown of red blood cells. The severity of symptoms can vary widely among individuals, depending on the specific type of membranopathy. These disorders are often inherited and can be diagnosed through blood tests that examine the shape and stability of red blood cells.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503271-24-01Protocol codeSATISFYEstimated enrolment33 patientsSponsorEurobloodnet Association

sourced from the EU Clinical Trials Register and site verification

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