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Study on the Effectiveness and Safety of Mitapivat for Patients with Non-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 non-transfusion-dependent alpha- or beta-thalassemia. Thalassemia is a blood disorder that affects the body's ability to produce hemoglobin, which is essential for carrying oxygen in the blood. People with this condition do not require regular blood transfusions. The study is testing a treatment called Mitapivat, which is taken in tablet form. Mitapivat is being compared to a placebo to see how effective it is in treating anemia, a condition where there is a lack of healthy red blood cells, in individuals with this type of thalassemia.

The purpose of the study is to evaluate the effectiveness and safety of Mitapivat in improving anemia in patients with non-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 several weeks, with regular check-ups to monitor the participants' health and response to the treatment.

Throughout the study, the main goal is to observe if there is an increase in hemoglobin levels, which would indicate an improvement in anemia. Participants will be closely monitored to ensure their safety and to gather data on how well Mitapivat works compared to the placebo. This research aims to provide valuable information that could lead to better treatment options for individuals with non-transfusion-dependent thalassemia.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization

    Upon joining the study, participants are randomly assigned to one of two groups. One group receives the active medication, mitapivat, while the other group receives a placebo. This process is double-blind, meaning neither the participants nor the researchers know who is receiving the active medication or the placebo.

  2. Step 2

    Medication administration

    Participants take the assigned medication orally in the form of a tablet. The specific dosage and frequency are determined by the study protocol and are administered consistently throughout the trial period.

  3. Step 3

    Monitoring and assessments

    Regular monitoring and assessments are conducted to evaluate the effect of the medication on anemia. This includes measuring hemoglobin levels to determine any changes in concentration. The primary goal is to observe a hemoglobin response, defined as an increase of at least 1.0 g/dL in average hemoglobin concentration from Week 12 through Week 24 compared with baseline.

  4. Step 4

    Completion of study

    The study is estimated to conclude by May 23, 2029. Participants continue to follow the study procedures until the end of the trial, ensuring all data is collected for analysis.

Who can join the trial?

7 criteria

  • You must be at least 18 years old when you agree to join the study.
  • You need to have a confirmed diagnosis of **thalassemia**, which is a blood disorder. This can be checked through specific tests like **Hb electrophoresis**, **Hb high-performance liquid chromatography**, or **DNA analysis**. If these tests are not in your medical records, they can be done during the study's screening period.
  • Your **hemoglobin (Hb) concentration** should be 10.0 g/dL or lower, based on at least two measurements taken at least 7 days apart during the screening period. Hemoglobin is a protein in your blood that carries oxygen.
  • You should not be dependent on blood transfusions, meaning you have received 5 or fewer red blood cell units in the 24 weeks before the study starts, and no transfusions 8 weeks before agreeing to join the study or during the screening period.
  • If you are taking **hydroxyurea**, a medication, your dose must not have changed for at least 16 weeks before the study starts.
  • 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 birth control, one of which must be highly effective, from the time you agree to join the study until 28 days after the last dose of the study drug. The second form can be a barrier method like a condom.
  • You must provide written 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?

6 criteria

  • Patients who need regular blood transfusions cannot participate. This means if you need to receive blood from someone else often, you are not eligible.
  • Patients with other serious health conditions that might interfere with the study cannot participate. This means if you have other major health problems, you might not be able to join.
  • Patients who are pregnant or breastfeeding cannot participate. This means if you are expecting a baby or nursing, you are not eligible.
  • Patients who are taking certain medications that could affect the study cannot participate. This means if you are on specific drugs that might change the study results, you cannot join.
  • Patients who have participated in another clinical trial recently cannot participate. This means if you have been part of another study not long ago, you are not eligible.
  • Patients who have a history of allergic reactions to the study medication cannot participate. This means if you have had bad reactions to the medicine being tested, you cannot join.
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Investigated drugs

Mitapivat is a medication being studied for its potential to treat anemia in patients with non-transfusion-dependent alpha or beta thalassemia. It works by activating an enzyme in red blood cells, which may help improve their function and increase hemoglobin levels, potentially reducing the symptoms of anemia.

What is already known about the treatment

Mitapivat – Mitapivat is administered orally in tablet form and is currently being studied in clinical trials for its effectiveness in treating non-transfusion-dependent alpha or beta thalassemia. This medication is in the investigational stage, with ongoing research to evaluate its safety and efficacy. It is primarily indicated for managing anemia associated with these types of thalassemia. Mitapivat works by activating an enzyme called pyruvate kinase, which plays a crucial role in energy production in red blood cells, helping to improve their function and lifespan. It is classified as a pyruvate kinase activator in pharmacology.

Investigated diseases

Non–Transfusion-Dependent Alpha- or Beta-Thalassemia – This is a genetic blood disorder characterized by reduced production of hemoglobin, the protein in red blood cells that carries oxygen. In this condition, individuals do not require regular blood transfusions to manage their symptoms. The disease results from mutations in the genes responsible for producing the alpha or beta chains of hemoglobin. As a result, affected individuals may experience mild to moderate anemia, which can lead to fatigue and weakness. The severity of symptoms can vary, and some individuals may have a normal life without significant health issues. The condition is typically identified through blood tests that reveal low hemoglobin levels and abnormal red blood cell shapes.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512745-16-00Protocol codeAG348-C-017Estimated enrolment172 patientsSponsorAgios Pharmaceuticals Inc.

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