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Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment

Verified siteInvestigationalNo 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 focuses on studying long-term treatment of sickle cell disease and thalassemia, which are inherited blood disorders that affect how red blood cells function in the body. The study uses etavopivat (also known as FT-4202), which is given as a 200 mg tablet taken by mouth. These conditions can cause severe pain, fatigue, and other serious health problems due to abnormal red blood cells.

The purpose of this research is to understand how safe and effective etavopivat is when used for an extended period in adults, teenagers, and children who have previously completed treatment with this medication in other studies. The medication will be given to participants who have shown improvement during their previous treatment with etavopivat.

During the study, patients may continue taking other medications they were already using for their condition, such as hydroxyurea, crizanlizumab, or L-glutamine, as long as their doses have remained stable. The study will monitor various aspects of the participants' health, including any side effects, hospital stays, and changes in their blood cell measurements over time.

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

    Initial qualification

    Your participation begins after completing a treatment period in another etavopivat study for sickle cell disease or thalassaemia

    The doctor will evaluate if you have received clinical benefits from the previous etavopivat treatment

    If you experienced any dose reductions or treatment interruptions, you will need to restart the treatment before entering this study

  2. Step 2

    Medication continuation

    You will continue taking etavopivat tablets (200 mg) by mouth

    If you are currently taking other medications such as hydroxyurea, crizanlizumab, or l-glutamine oral powder, you may continue them if you have been taking them at a stable dose

  3. Step 3

    Long-term monitoring

    The doctor will monitor your health status throughout the study period

    Regular checks will include measuring your hemoglobin levels (a protein that carries oxygen in your blood)

    Any hospital stays will be recorded

    If needed, the number of blood transfusions you receive will be tracked

  4. Step 4

    Safety assessment

    The doctor will monitor and record any side effects you may experience

    The frequency of pain episodes related to your condition will be tracked

    The study will continue until November 2029

Who can join the trial?

8 criteria

  • Must provide informed consent before participating in any study activities
  • Must be currently participating in another study using etavopivat (a medication) for treating Sickle Cell Disease or Thalassemia, and have completed at least one treatment period
  • Must have shown positive results from etavopivat treatment, as determined by the doctor
  • If the medication dose was reduced or stopped temporarily, patients must try the medication again before joining this study
  • Patients taking other medications such as hydroxyurea, crizanlizumab, or l-glutamine oral powder may participate if:
    • They have been on a stable dose during their previous study (only changes due to weight or age were allowed)
    • They have been taking the medication as prescribed during their previous study participation
    • Both male and female patients can participate
    • The study includes children, adolescents, and adults

Who cannot join the trial?

12 criteria

  • Patients who have not previously participated in studies with etavopivat (the study medication)
  • Individuals under 2 years of age
  • Pregnant or breastfeeding women
  • Patients with severe liver problems or impaired kidney function
  • Individuals with uncontrolled high blood pressure
  • Patients with active, uncontrolled infections
  • People with a history of severe allergic reactions to medications
  • Patients taking certain medications that might interact with the study drug
  • Individuals participating in other clinical trials at the same time
  • Patients who have had major surgery within the past 30 days
  • People with significant heart conditions or abnormal heart rhythm
  • Patients with unstable medical conditions that could interfere with the study
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Investigated drugs

Etavopivat is a medication being studied for treating blood disorders, specifically sickle cell disease and thalassemia. It works by modifying how red blood cells function to help improve their shape and survival in people with these conditions. This medication is being tested as a long-term treatment option for adults, adolescents, and children who have previously participated in other studies using this same drug.

What is already known about the treatment

Etavopivat - An oral medication under clinical investigation for treating Sickle Cell Disease and Thalassemia, administered in tablet form. This drug works by modifying the function of pyruvate kinase-R enzyme in red blood cells, which helps improve cellular metabolism and reduce sickling of red blood cells. Currently undergoing clinical trials to evaluate its long-term safety and effectiveness, etavopivat represents a novel therapeutic approach in hematological disorders, particularly focusing on patients ranging from children to adults. The medication belongs to the class of pyruvate kinase activators and is being studied for its potential to provide a sustained improvement in patients who have completed previous etavopivat treatment courses.

Investigated diseases

  • Sickle Cell Disease

    A genetic blood disorder where red blood cells become crescent-shaped instead of round. These abnormal cells can get stuck in blood vessels, causing pain and blocking blood flow. The condition is present from birth and affects how oxygen is delivered throughout the body. The altered blood cells break down faster than normal ones, leading to anemia.

  • Thalassemia

    A hereditary blood condition where the body makes an abnormal form or inadequate amount of hemoglobin. The disorder leads to the destruction of red blood cells, resulting in anemia. There are different types of thalassemia, varying in severity depending on the specific genetic changes involved. The condition affects the body's ability to produce normal adult hemoglobin, which is essential for carrying oxygen throughout the body.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-510805-27-00Protocol codeNN7535-7822Estimated enrolment474 patientsSponsorNovo Nordisk A/S

sourced from the EU Clinical Trials Register and site verification

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