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Study on the Effects of Etavopivat for Patients Aged 12-65 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 study is testing a new treatment called Etavopivat, which is a type of medication known as a pyruvate kinase activator. This medication is being tested in two different doses, 100 mg and 200 mg, and will be compared to a placebo. The purpose of the study is to see if Etavopivat can help improve hemoglobin levels and reduce the number of painful episodes known as vaso-occlusive crises, which are common in people with sickle cell disease.

Participants in the study will be randomly assigned to receive either Etavopivat 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 not biased. The study will take place over a period of time, with regular check-ins to monitor the participants' health and any changes in their condition. The main goal is to assess the effectiveness of Etavopivat in improving health outcomes for those with sickle cell disease.

Throughout the study, participants will have their hemoglobin levels checked and will be monitored for any changes in their condition. The study will also look at other health markers related to sickle cell disease, such as the number of red blood cells and certain proteins in the blood. By the end of the study, researchers hope to determine whether Etavopivat is a safe and effective treatment option for people living 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, consent is required. A confirmed diagnosis of sickle cell disease is necessary.

    Eligibility includes having 2-15 episodes of vaso-occlusive crises in the past year and specific hemoglobin levels during screening.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current treatments.

    Patients on hydroxyurea must have a stable dose for at least 90 days before starting the study treatment.

  3. Step 3

    Treatment phase

    The treatment involves taking etavopivat or a placebo orally. Dosages include 100 mg or 200 mg tablets.

    The treatment is administered over a 52-week period, with regular monitoring of hemoglobin levels and vaso-occlusive crisis rates.

  4. Step 4

    Monitoring and evaluation

    Primary evaluations occur at Week 24 and Week 52, focusing on hemoglobin response and annualized vaso-occlusive crisis rates.

    Secondary evaluations include changes in clinical laboratory measurements and patient-reported fatigue levels.

  5. Step 5

    Completion of study

    The study is estimated to conclude by February 2027. Final assessments will be conducted to evaluate the overall efficacy of the treatment.

Who can join the trial?

7 criteria

  • Provide consent to participate in the study.
  • Have a confirmed diagnosis of sickle cell disease (SCD).
  • Have experienced 2 to 15 episodes of documented vaso-occlusive crises in the past 12 months. A vaso-occlusive crisis is a painful episode that occurs when blood flow is blocked by sickle-shaped cells.
  • Have a hemoglobin level between 5.5 and 10.5 g/dL during screening. Hemoglobin is a protein in red blood cells that carries oxygen.
  • If taking hydroxyurea, must have been on a stable dose for at least 90 days before starting the study treatment. Hydroxyurea is a medication used to reduce complications of sickle cell disease.
  • Female patients who can have children must use acceptable methods of contraception. Male patients must be willing to use acceptable methods of contraception.
  • Patients on crizanlizumab or L-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose for at least 12 months, have been at least 80% compliant with the planned regimen during the 12 months prior to consent, and meet the vaso-occlusive crisis eligibility requirement.

Who cannot join the trial?

5 criteria

  • Individuals who do not have sickle cell disease (SCD) cannot participate. SCD is a group of inherited red blood cell disorders.
  • Participants must be within the specified age range for the study.
  • Both males and females are eligible, but certain conditions may exclude participation.
  • Individuals who are part of a vulnerable population may be excluded. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.
  • Any other specific medical conditions or treatments that might interfere with the study's objectives could lead to exclusion.
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Investigated drugs

Etavopivat is a medication being studied for its potential benefits in patients with Sickle Cell Disease. It works by activating an enzyme called pyruvate kinase, which may help improve the levels of hemoglobin in the blood. This could lead to better oxygen transport in the body and potentially reduce the frequency of painful episodes known as vaso-occlusive crises, which are common in people with Sickle Cell Disease.

What is already known about the treatment

Etavopivat – Etavopivat is administered orally and is currently being studied in clinical trials for its potential use in treating sickle cell disease (SCD). It is a pyruvate kinase activator, which means it works by enhancing the activity of the pyruvate kinase enzyme, leading to improved energy production in red blood cells. This action helps to increase hemoglobin levels and reduce the frequency of vaso-occlusive crises, which are painful episodes common in SCD. As a pharmacological agent, Etavopivat is classified as a metabolic modulator, and its efficacy is being compared to a placebo in ongoing research.

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, the 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 vaso-occlusive crises, and other complications. Over time, the disease can cause damage to organs and tissues due to reduced blood flow.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-511535-97-00Protocol code4202-HEM-301Estimated enrolment450 patientsSponsorNovo Nordisk A/S

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