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Study on the Effects of Mitapivat for Patients with Transfusion-Dependent Alpha or Beta Thalassemia

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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 a condition known as transfusion-dependent thalassemia, which includes both alpha-thalassemia and beta-thalassemia. These are blood disorders that affect the body's ability to produce normal hemoglobin, leading to a need for regular blood transfusions. The study is testing a treatment called Mitapivat, also known by its code name AG-348, which is taken in tablet form. Mitapivat is being compared to a placebo to see how it affects the need for blood transfusions in people with this condition.

The purpose of the study is to evaluate how effective and safe Mitapivat is for individuals with transfusion-dependent thalassemia. Participants in the study will be randomly assigned to receive either Mitapivat or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are unbiased. The study will take place over a period of time, during which participants will be monitored to see if there is a reduction in the number of blood transfusions they need.

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment. The main goal is to see if Mitapivat can reduce the number of blood transfusions needed by at least 50% over a 12-week period compared to the start of the study. This could potentially improve the quality of life for those living with transfusion-dependent thalassemia by reducing their reliance on blood transfusions.

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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical records for a documented diagnosis of thalassemia and ensuring transfusion dependency.

    If necessary, DNA analysis may be performed to confirm the diagnosis. This can be done by a local laboratory or the study's central laboratory if local facilities are unavailable.

  2. Step 2

    Randomization

    Participants are randomly assigned to receive either mitapivat or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

  3. Step 3

    Treatment phase

    During the treatment phase, participants take the assigned medication orally in the form of a tablet. The specific dosage and frequency are determined by the study protocol.

    The treatment continues for a specified duration, with regular monitoring to assess the effect on transfusion needs.

  4. Step 4

    Monitoring and evaluation

    Throughout the study, participants undergo regular evaluations to monitor their health and the effectiveness of the treatment. This includes tracking the number of red blood cell transfusions required.

    The primary goal is to achieve a transfusion reduction response, defined as a 50% reduction in transfused red blood cell units over any consecutive 12-week period through Week 48 compared to baseline.

  5. Step 5

    Study completion

    At the end of the study period, participants complete a final assessment to evaluate the overall impact of the treatment.

    The study is estimated to conclude by October 20, 2029.

Who can join the trial?

6 criteria

  • You must be **18 years or older** at the time you agree to participate in the study.
  • You need to have a **documented diagnosis of thalassemia**. This includes different types like beta-thalassemia, alpha-thalassemia, or HbH disease. This diagnosis should be based on a DNA test from your medical records. If this test is not available, it can be done during the study's screening period.
  • You must be **transfusion dependent**, meaning you have received between 6 to 20 units of red blood cells and have not gone more than 6 weeks without a transfusion in the 24 weeks before starting the study.
  • If you are taking **hydroxyurea** (a medication), your dose must have been stable for at least 16 weeks before starting the study.
  • If you are a woman who can become pregnant, you must either not engage in sexual activities that could lead to pregnancy or agree to use two forms of **contraception** (birth control) during the study and for 28 days after the last dose of the study drug. One form must be highly effective, and the other can be a barrier method like a condom.
  • You must provide **written informed consent** before any study-related procedures and be willing to follow all study procedures for the entire duration of the study.

Who cannot join the trial?

8 criteria

  • Patients who are not dependent on blood transfusions for their condition.
  • Patients with medical conditions other than **Alpha- or Beta-Thalassemia** that require regular blood transfusions.
  • Patients who are not within the specified age range for the study.
  • Patients who are not able to follow the study procedures or take the study medication as required.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients with certain other health conditions that might interfere with the study.
  • Patients who are unable to provide informed consent or do not have a legal representative to do so.
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Investigated drugs

Mitapivat is a medication being studied for its potential to reduce the need for blood transfusions in people with transfusion-dependent alpha or beta thalassemia. Thalassemia is a blood disorder that affects the production of hemoglobin, leading to anemia and the need for regular blood transfusions. Mitapivat works by activating an enzyme in red blood cells, which may help improve their function and reduce the frequency of transfusions needed by patients.

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 potential use in treating transfusion-dependent alpha- or beta-thalassemia. The main therapeutic indication for mitapivat is to reduce the transfusion burden in patients with these types of thalassemia. At the molecular level, mitapivat works by activating the enzyme pyruvate kinase, which plays a crucial role in the energy production process of red blood cells. This medication is classified as a pyruvate kinase activator.

Investigated diseases

Transfusion-Dependent Alpha- or Beta-Thalassemia – This is a genetic blood disorder characterized by the body's inability to produce enough hemoglobin, the protein in red blood cells that carries oxygen. In alpha-thalassemia, there is a defect in the alpha globin chain, while in beta-thalassemia, the defect is in the beta globin chain. Individuals with this condition often require regular blood transfusions to manage symptoms and maintain adequate hemoglobin levels. Over time, the need for transfusions can lead to complications such as iron overload in the body. The disease is typically diagnosed in early childhood and requires ongoing medical management. Symptoms can include fatigue, weakness, and pale skin due to anemia.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512747-23-00Protocol codeAG348-C-018Estimated enrolment204 patientsSponsorAgios Pharmaceuticals Inc.

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