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A study of etavopivat to reduce vaso‑occlusive crises in children 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

Sickle cell disease is a genetic blood condition that makes red blood cells stiff and shaped like a sickle, leading to painful episodes called VOC (blockages in small blood vessels). The study tests an oral medicine called etavopivat, which is provided in granule or tablet form, to see if it can reduce these painful episodes in children.

The purpose of the study is to determine whether etavopivat can lower the number of VOC events compared with placebo. Children are randomly assigned to receive either the study medicine or a matching placebo, and neither the participants nor the doctors know which one is being taken (double‑blind). The medication is taken by mouth each day for several months, with regular clinic visits for safety checks and blood tests that measure hemoglobin (the protein that carries oxygen), lactate dehydrogenase (an enzyme that rises when red cells break down), reticulocyte count (the number of young red cells), and bilirubin (a waste product from broken red cells). Information on hospital stays, emergency visits, and days missed from school is also collected, and at the end the frequency of painful episodes is compared between the two groups.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial study visit

    At the first visit, eligibility is confirmed and baseline information is collected. this includes a review of your medical history, blood tests, and questionnaires about your health and daily activities.

    The purpose of these assessments is to establish a reference point for later comparison while you are participating in the study for sickle cell disease.

  2. Step 2

    Random assignment to treatment

    After the baseline visit, you are randomly assigned to receive either the study drug etavopivat or a matching placebo. the study is double‑blind, so neither you nor the study staff know which one you receive.

  3. Step 3

    Starting the medication

    You will begin taking an oral medication each day as instructed by the study team.

    If you are assigned to the study drug, the form may be granules (etavopivat b 10041 or etavopivat b 10042) or a tablet (etavopivat c 10164). the label shows a dose of 00 mg, taken by mouth.

    If you are assigned to the placebo, you will take a matching oral preparation that does not contain active medication.

  4. Step 4

    Regular study visits

    You will attend scheduled visits throughout the trial. during each visit, blood samples are taken to check safety and effectiveness, and you complete questionnaires about pain, fatigue, and daily activities.

    The study staff also ask about any episodes of vaso‑occlusive crisis (voc), which are painful events that can require medical attention.

  5. Step 5

    Ongoing monitoring of outcomes

    Throughout the trial, the number of voc events, hospital stays, emergency‑room visits, missed school days, and fatigue levels are recorded.

    You will be asked to report any new symptoms, side effects, or changes in your health between visits.

  6. Step 6

    Completion of treatment period

    After the predetermined treatment period ends, you stop taking the study medication.

    A final set of assessments, including blood tests and questionnaires, is performed to evaluate the overall effect of the treatment.

  7. Step 7

    Final follow‑up visit

    A follow‑up visit is scheduled after treatment ends to check for any delayed effects and to ensure safety.

    The study team reviews all collected data and concludes your participation in the trial.

Who can join the trial?

7 criteria

  • Informed consent must be given before any study‑related activities are performed.
  • The child can be male or female.
  • The child must be between 2 and 11 years old at the time of randomisation.
  • The child must have a confirmed diagnosis of sickle cell disease (SCD) with documentation of the specific type (such as HbSS, HbSC, HbSβ0‑thalassaemia, HbSβ+ thalassemia, or other variants) from previous laboratory testing. Laboratory tests may include Hb electrophoresis, high‑performance liquid chromatography, or similar methods that examine the blood’s hemoglobin.
  • The child’s hemoglobin level must be at least 5.5 g/dL and no more than 10.5 g/dL at the screening visit. Hemoglobin is the protein in red blood cells that carries oxygen.
  • The child must have had at least 2 and no more than 15 documented vaso‑occlusive crises (VOC) in the 12 months before screening, with records in the medical chart. VOC are painful episodes caused by sickle cells blocking blood flow.
  • The child must meet any other requirements that are described in the study protocol.

Who cannot join the trial?

12 criteria

  • Allergic reaction (known or suspected) to the study drug or any similar medication.
  • Other reasons listed in the detailed study plan that may prevent participation.
  • Body weight less than 10 kilograms (about 22 pounds).
  • Any serious liver problem, such as:
    • Blood tests showing ALT or AST levels more than three times the normal range (these are enzymes that indicate liver injury).
    • Direct bilirubin level more than twice the normal range (bilirubin is a substance processed by the liver).
    • Diagnosed liver disease with scarring called cirrhosis or severe fibrosis.
    • Severe kidney problems, meaning an estimated glomerular filtration rate (eGFR) less than 30 mL/min/1.73 m² (a measure of kidney function) or being on long‑term dialysis.
    • History of a clear stroke, bleeding inside the brain (intracranial haemorrhage), or serious blood‑vessel disease in the brain seen on a special scan called magnetic resonance angiography.
    • Iron deficiency (low blood iron or ferritin below 10 µg/L) if the person cannot take iron supplements during the study.
    • Deficiency of folate (vitamin B9) or vitamin B12 if the person cannot take the appropriate supplements during the study.
    • Inability or unwillingness to use malaria prevention methods (such as bed nets or recommended medicines) if living in an area where malaria is common.
    • Use of medicines that strongly increase the activity of liver enzymes called CYP3A4/5 within two weeks before starting the study or a need to use such medicines during the study.
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Investigated drugs

  • etavopivat

    (oral granules) is an investigational medicine being studied to help children with sickle cell disease. It is taken by mouth in a granule form that dissolves in the mouth or a drink. The drug works by improving how red blood cells produce and use energy, which can make them less likely to become stiff and stick together. By doing this, etavopivat may lower the number of painful episodes called vaso‑occlusive crises that children with sickle cell disease often experience.

  • etavopivat

    (tablet) is the same investigational medicine but provided as a tablet that is swallowed. Like the granule version, it is taken orally and aims to improve red blood cell energy metabolism. The goal is to reduce the frequency of painful vaso‑occlusive crises in children with sickle cell disease, helping them feel better and have fewer hospital visits.

What is already known about the treatment

Etavopivat - Etavopivat is taken by mouth as either granules or tablets and is given to children with sickle cell disease. It is an orphan‑drug currently being studied in clinical trials and has not yet been approved for general medical use. The medication works by activating the enzyme pyruvate kinase‑R in red blood cells, which raises cellular energy (ATP) and helps keep the cells from sickling, thereby lowering the number of painful vaso‑occlusive crises. It is classified as a pyruvate kinase activator, a small‑molecule oral therapy aimed at improving red blood cell function in sickle cell disease.

Investigated diseases

Sickle cell disease - Sickle cell disease is a genetic condition that affects the blood’s red cells, causing them to become stiff and shaped like a crescent. These abnormal cells can block small blood vessels, leading to episodes of pain and reduced blood flow. Over time, the repeated blockages can cause damage to organs such as the spleen, kidneys, and bones. The disease often results in frequent pain crises, anemia, and increased need for medical care. Children may experience growth delays and fatigue as the condition progresses.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2025-524464-38-00Protocol codeNN7535-8365Estimated enrolment260 patientsSponsorNovo Nordisk A/S

sourced from the EU Clinical Trials Register and site verification

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