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A study to test how etavopivat works and its safety in children with sickle cell disease

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 study is looking at sickle cell disease, a condition that affects the shape of red blood cells and can cause various health problems. The study will test a medicine called etavopivat in children and young people from 6 months to under 18 years of age. Etavopivat is given by mouth in the form of granules. The medicine is being tested to see how it works in the body and whether it is safe for children with sickle cell disease.

The purpose of the study is to understand how the body handles etavopivat and to check for any unwanted effects during a 24-week treatment period. During the study, doctors will measure how much medicine is in the blood after a single dose and during regular treatment. They will also monitor for any side effects and record how often treatment needs to be stopped or changed. The study will look at whether the medicine helps increase the level of a substance in the blood called hemoglobin, which carries oxygen throughout the body. Doctors will also track painful episodes called vaso-occlusive crises, which are common in sickle cell disease when blood flow becomes blocked.

Participants will be divided into different groups based on their age, starting with older children and then including younger ones. Some children in the study may already be taking other treatments for sickle cell disease, such as hydroxyurea, crizanlizumab, or L-glutamine, and can continue these medicines if their dose has been stable. The study will also measure changes in tiredness levels and check blood flow in the brain using a test called transcranial Doppler ultrasonography in children over 2 years old. After the first 24 weeks, there will be an extension period where the medicine can continue to be given while doctors keep checking for safety.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment period begins

    Upon joining the study, treatment with etavopivat will begin. This medication is provided as granules that are taken by mouth.

    The specific dose of etavopivat will be determined based on individual factors. The medication is taken regularly as prescribed by the study doctor.

    If currently taking hydroxyurea (a medication for sickle cell disease), the same dose will be continued without changes throughout the study.

    If currently taking crizanlizumab or L-glutamine (other treatments for sickle cell disease), these medications will be continued at the same dose.

  2. Step 2

    Blood sample collection for medication levels

    Blood samples will be collected to measure how the body processes etavopivat. This includes measuring the maximum concentration of the medication in the blood and how long it stays in the body.

    These measurements help determine if the medication dose is appropriate for the individual patient.

  3. Step 3

    Week 12 assessment

    At 12 weeks after starting treatment, several assessments will be performed.

    Hemoglobin levels (a protein in red blood cells that carries oxygen) will be measured through a blood test to see if there has been an increase from the starting level.

    A questionnaire about fatigue (tiredness) may be completed if the patient is between 5 and 18 years of age. This questionnaire helps assess changes in energy levels.

    The study doctor will review any side effects or health changes that have occurred.

  4. Step 4

    Week 24 assessment and completion of primary treatment period

    At 24 weeks after starting treatment, the primary treatment period ends.

    The same assessments performed at Week 12 will be repeated, including hemoglobin levels and the fatigue questionnaire (if applicable).

    For patients older than 2 years of age, a transcranial Doppler ultrasonography may be performed. This is a painless test that uses sound waves to measure blood flow in the brain.

    Throughout the 24-week period, the study doctor will monitor for vaso-occlusive crises (painful episodes caused by blocked blood flow, a common complication of sickle cell disease). The number and frequency of these episodes will be recorded.

  5. Step 5

    Extension period (optional)

    After completing the 24-week primary treatment period, there may be an option to continue in an extension period of the study.

    During this extension period, etavopivat treatment will continue, and the study doctor will continue to monitor for any side effects or health changes.

    The extension period allows for longer-term observation of the medication's effects and safety.

  6. Step 6

    End of treatment

    When treatment with etavopivat is stopped, either after the primary treatment period or after the extension period, a final assessment will be conducted.

    Follow-up will continue for 90 days after the last dose of etavopivat to monitor for any delayed effects.

    Female patients of childbearing age should not donate eggs, and male patients should not donate sperm during this 90-day period after stopping the medication.

Who can join the trial?

8 criteria

  • The patient's parent, legal guardian, or legal representative must provide written permission for the patient to participate, and the patient must agree to take part in a way that is appropriate for their age
  • The patient must be older than 6 months and younger than 18 years of age at the time of joining the study
  • The patient must have a confirmed diagnosis of Sickle Cell Disease, which is a blood disorder where red blood cells become abnormally shaped. The specific type of Sickle Cell Disease must be documented through previous laboratory tests
  • The patient's hemoglobin level, which is the protein in red blood cells that carries oxygen, must be between 5.5 and 10.5 grams per deciliter
  • The patient must have severe Sickle Cell Disease, shown by at least one of the following: having had between 2 and 15 pain crisis episodes in the past 12 months that are documented in medical records, being hospitalized for any Sickle Cell Disease complication in the past 12 months, having protein in the urine indicating early kidney problems, or having had abnormal blood flow speed in brain blood vessels in the past 12 months but not currently receiving regular blood transfusions
  • If the patient is taking hydroxyurea, which is a medication used to treat Sickle Cell Disease, the dose must have been stable for at least 90 days before starting the study, with no more than a 20% change in dosing, and no expected need for dose changes during the study
  • If the patient is taking crizanlizumab, a medication that helps prevent pain crises, or L-glutamine, an amino acid supplement, they must have been on a stable dose for at least 12 months. For patients on crizanlizumab, they must have taken at least 80% of their planned doses during the past 12 months
  • Female patients who are able to become pregnant must use acceptable birth control methods and agree not to donate eggs from the start of the study until 90 days after the last dose of study medication. Male patients must use acceptable birth control methods and agree not to donate sperm from the start of the study until 90 days after the last dose of study medication

Who cannot join the trial?

7 criteria

  • The study does not list specific exclusion criteria in the provided information, meaning detailed reasons why patients cannot participate are not available in this data
  • Generally, clinical trials may exclude patients based on factors such as other serious health conditions, certain medications they are taking, or specific laboratory test results that fall outside acceptable ranges
  • Patients who are unable to follow the study requirements or attend scheduled visits may not be eligible
  • Women who are pregnant or breastfeeding (nursing a baby) are typically excluded from medication studies
  • Patients with severe kidney disease (problems with organs that filter waste from blood) or liver disease (problems with the organ that processes medications) may not be able to participate
  • Those who have participated in another research study recently may be excluded
  • Patients with known allergies (bad reactions) to the study medication or similar drugs may not qualify
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Investigated drugs

Etavopivat is an investigational medication being studied for the treatment of sickle cell disease. This medicine works by helping red blood cells function better and may reduce some of the complications associated with sickle cell disease. In this study, researchers want to learn how the body processes this medication in children and teenagers with sickle cell disease, and whether it is safe for them to take.

What is already known about the treatment

Etavopivat – Etavopivat is an investigational oral medication currently being studied in clinical trials for the treatment of Sickle Cell Disease. This drug works at the molecular level by activating an enzyme called pyruvate kinase in red blood cells, which helps these cells maintain their energy levels and prevents them from taking on the abnormal sickle shape that causes complications in patients with this condition. The medication is being evaluated for its safety, tolerability, and how the body processes it (pharmacokinetics) in pediatric patients over a 24-week treatment period. Etavopivat belongs to a class of drugs known as pyruvate kinase activators, which are designed to improve red blood cell function and reduce disease-related complications.

Investigated diseases

Sickle Cell Disease – Sickle Cell Disease is a genetic blood disorder that affects the shape and function of red blood cells. In this condition, red blood cells become rigid and shaped like crescents or sickles instead of being round and flexible. These abnormally shaped cells can block blood flow through small vessels, leading to episodes of pain called vaso-occlusive crises. The sickled cells also break down more quickly than normal red blood cells, causing a shortage of red blood cells in the body, known as anemia. This ongoing destruction of red blood cells can lead to fatigue and weakness. The disease is inherited and affects the hemoglobin, which is the protein in red blood cells that carries oxygen throughout the body.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase ITrial ID2024-519881-32-00Protocol code4202-HEM-202Estimated enrolment95 patientsSponsorNovo Nordisk A/S

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