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Study on the Effectiveness and Safety of Mitapivat for Patients with Sickle Cell Disease

Fast replyInvestigationalNo 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 effects of a medication called Mitapivat on individuals with Sickle Cell Disease. Sickle Cell Disease is a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health issues, including anemia and pain crises. The purpose of this study is to evaluate how effective and safe Mitapivat is in treating these symptoms.

Participants in the study will be randomly assigned to receive either Mitapivat or a placebo, which is a substance with no active medication. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are unbiased. The study will take place over a period of time, during which participants will take the medication orally in the form of tablets.

The main goal is to see if Mitapivat can improve anemia by increasing hemoglobin levels, which is a protein in red blood cells that carries oxygen. Additionally, the study will look at how Mitapivat affects the frequency of sickle cell pain crises. Participants will be monitored regularly to assess any changes in their condition and to ensure their safety throughout the study. The results will help determine if Mitapivat is a beneficial treatment option for people 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, the participant will be randomly assigned to receive either mitapivat or a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.

    The study is designed to evaluate the effectiveness and safety of mitapivat in individuals with sickle cell disease.

  2. Step 2

    Medication administration

    Participants will take mitapivat or placebo orally in the form of tablets. The tablets are available in different dosages: 5 mg, 20 mg, 50 mg, or 100 mg.

    The specific dosage and frequency of administration will be determined by the study protocol and communicated to the participant at the start of the trial.

  3. Step 3

    Monitoring and assessments

    Throughout the study, participants will undergo regular monitoring to assess the impact of the treatment on anemia and sickle cell pain crises.

    Key assessments include measuring hemoglobin levels and tracking the frequency of pain crises. These assessments will occur from Week 24 through Week 52.

  4. Step 4

    Primary and secondary outcomes

    The primary outcomes of the study are an increase in hemoglobin levels and a reduction in the annual rate of sickle cell pain crises.

    Secondary outcomes include changes in bilirubin levels, reticulocyte percentage, fatigue scores, and hospitalization frequency due to pain crises.

  5. Step 5

    Completion of the study

    The study is expected to conclude by April 2030. Participants will be informed of their treatment group and the overall results of the study upon its completion.

    Participants are required to comply with all study procedures and attend all scheduled visits throughout the duration of the study.

Who can join the trial?

7 criteria

  • Must be at least 16 years old. If you are 16 or 17, you need to be at a certain stage of physical development called **Tanner Stage 5**.
  • Must have a confirmed diagnosis of **sickle cell disease (SCD)**, which includes different types like HbSS, HbSC, HbS/β0-thalassemia, HbS/β+-thalassemia, or other similar conditions.
  • Must have experienced at least 2 but no more than 10 **sickle cell pain crises (SCPCs)** in the past year.
  • Must have a **hemoglobin** level between 5.5 and 10.5 grams per deciliter, based on an average of at least 2 tests taken at least 7 days apart during the screening period. Hemoglobin is a protein in your blood that carries oxygen.
  • If you are taking **hydroxyurea** (a medication for SCD), your dose must not have changed for at least 90 days before starting the study. If you stop taking it, you need to wait 90 days before joining the study.
  • Women who can become pregnant must either not engage in sexual activities that could lead to pregnancy or agree to use 2 forms of birth control, one of which must be very effective, from the time they 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.
  • Must provide written consent to participate in the study. If under 18, parental permission and the child's agreement are required. Participants must be willing to follow all study procedures for the entire duration of the study.

Who cannot join the trial?

6 criteria

  • Individuals who do not have sickle cell disease (SCD) cannot participate. Sickle cell disease is a group of inherited red blood cell disorders.
  • Individuals who do not experience anemia related to sickle cell disease cannot participate. Anemia is a condition where you don't have enough healthy red blood cells to carry oxygen to your body's tissues.
  • Individuals who do not have sickle cell pain crises (SCPCs) cannot participate. These are episodes of pain that occur in people with sickle cell disease.
  • Individuals who are not within the specified age range for the study cannot participate. The study includes certain age groups, but the specific ages are not listed here.
  • Both male and female individuals are eligible, so exclusion is not based on gender.
  • Individuals who are not considered part of a vulnerable population may be excluded. Vulnerable populations include groups that may need special protection or care.
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Investigated drugs

Mitapivat is a medication being studied for its potential to help people with sickle cell disease. It is being tested to see if it can improve anemia, which is a condition where the blood doesn't have enough healthy red blood cells. The study is also looking at whether mitapivat can reduce the number of painful episodes, known as sickle cell pain crises, that people with sickle cell disease experience.

What is already known about the treatment

Mitapivat – Mitapivat is administered orally and is currently being studied in clinical trials for its potential use in treating anemia in individuals with sickle cell disease. It is in the Phase 2/3 stage of clinical trials, indicating that it is being evaluated for both efficacy and safety. The main therapeutic indication for mitapivat is to address anemia and potentially reduce sickle cell pain crises in patients with sickle cell disease. At the molecular level, mitapivat works by activating the enzyme pyruvate kinase, which plays a crucial role in the energy production pathway of red blood cells, thereby improving their function and survival. It is classified pharmacologically as a pyruvate kinase activator.

Investigated diseases

Anemia in Sickle Cell Disease – This condition occurs when there is a deficiency of red blood cells or hemoglobin in individuals with sickle cell disease. Sickle cell disease is a genetic disorder that causes red blood cells to become misshapen and break down. As a result, individuals experience chronic anemia, which can lead to fatigue and weakness. The disease progresses with episodes of pain, known as sickle cell pain crises, due to blocked blood flow. Over time, the anemia can affect various organs and lead to complications. The severity and frequency of symptoms can vary among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-515202-84-00Protocol codeAG348-C-020Estimated enrolment276 patientsSponsorAgios Pharmaceuticals Inc.

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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.

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