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Study on How Well Etavopivat Works for Adolescents and Adults 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 sickle cell disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study will evaluate the effectiveness and safety of a treatment called etavopivat, also known by its code name FT-4202. Participants in the study will receive either etavopivat or a placebo, which is a substance with no active medication.

The purpose of the study is to determine if etavopivat is more effective than a placebo in treating adolescents and adults with sickle cell disease. Participants will take part in a series of assessments over the course of the study, which will last for a period of time. These assessments will help researchers understand how well the treatment works and its safety profile. The study will involve regular check-ups and monitoring to track any changes in the participants' health and symptoms.

Throughout the study, researchers will pay close attention to the number of painful episodes known as vaso-occlusive crises, which are common in sickle cell disease. They will also monitor changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen, and other health indicators. The goal is to see if etavopivat can reduce these painful episodes and improve overall health outcomes for those 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

    Randomization and initial assessment

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

    An initial assessment is conducted to confirm eligibility, including a review of medical history and a physical examination.

  2. Step 2

    Treatment phase

    Participants take the assigned treatment orally in the form of a tablet. The dosage is 200 mg of etavopivat or a matching placebo.

    The treatment is administered daily for the duration of the study.

  3. Step 3

    Monitoring and follow-up

    Regular follow-up visits are scheduled to monitor health and response to the treatment. These visits include blood tests to measure haemoglobin levels and other relevant markers.

    Participants are assessed for any vaso-occlusive crises (VOCs), which are painful episodes common in sickle cell disease.

  4. Step 4

    Evaluation of outcomes

    The primary outcome is the number of VOC events requiring medical contact.

    Secondary outcomes include changes in haemoglobin levels, fatigue scores, and physical performance as measured by the 6-minute walking test.

  5. Step 5

    Completion and final assessment

    At the end of the study, a final assessment is conducted to evaluate the overall health and any changes experienced during the trial.

    Participants are informed of the treatment they received after the study concludes.

Who can join the trial?

5 criteria

  • Must be either male or female.
  • Must be at least 12 years old at the time of signing the informed consent.
  • Must have a confirmed diagnosis of sickle cell disease (SCD). This means having a specific type of blood disorder, which should be documented through previous lab tests or a screening test.
  • Must have experienced between 2 to 15 episodes of vaso-occlusive crises (VOCs) in the past year. These are painful events caused by blocked blood flow. The episodes must be recorded in the medical records before joining the study. Memories of events without documentation do not count.
  • Must have a hemoglobin level between 5.0 and 10.0 grams per deciliter (g/dL) at the time of screening. Hemoglobin is a protein in red blood cells that carries oxygen.

Who cannot join the trial?

3 criteria

  • Patients who do not have sickle cell disease cannot participate. Sickle cell disease is a condition where red blood cells, which carry oxygen, are shaped like a sickle or crescent instead of being round.
  • Patients who are not adolescents or adults are excluded. This means the study is only for people who are teenagers or older.
  • Patients who are not able to give their own consent or are considered part of a vulnerable group may not be eligible. A vulnerable group might include people who need extra protection, like those with certain disabilities or conditions.
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Investigated drugs

Etavopivat is a medication being studied for its potential to help people with sickle cell disease. Sickle cell disease is a condition where red blood cells become misshapen and can cause pain and other serious health problems. Etavopivat is being tested to see if it can improve the health of these red blood cells, making them more flexible and less likely to cause blockages in blood vessels. This study aims to determine if etavopivat is more effective than a placebo in treating adolescents and adults with sickle cell disease.

What is already known about the treatment

Etavopivat – Etavopivat is administered orally in the form of tablets. It is currently being studied in a phase 3 clinical trial to assess its effectiveness and safety for treating sickle cell disease in adolescents and adults. The main therapeutic indication for etavopivat is to improve the health of individuals with sickle cell disease by increasing the oxygen-carrying capacity of red blood cells. At the molecular level, etavopivat works by activating specific enzymes that help red blood cells maintain their normal shape and function, reducing the sickling process. It is classified pharmacologically as a hemoglobin modulator.

Investigated diseases

Sickle Cell Disease – Sickle cell disease is a genetic disorder that affects the red blood cells, causing them to become rigid and shaped like a crescent or sickle. These abnormally shaped cells can block blood flow in small blood vessels, leading to pain and potential organ damage. The disease is characterized by episodes of pain, known as vaso-occlusive crises, which can vary in frequency and severity. Over time, sickle cell disease can lead to complications such as anemia, increased risk of infections, and damage to organs like the spleen and liver. The condition is inherited and is most common in individuals of African, Mediterranean, or Middle Eastern descent. Regular medical care and monitoring are essential to manage symptoms and prevent complications.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-509175-16-00Protocol codeNN7535-7807Estimated enrolment408 patientsSponsorNovo Nordisk A/S

sourced from the EU Clinical Trials Register and site verification

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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.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).