AuthorisedTherapeutic confirmatory (Phase III)Interventional

A global phase 3 randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in children with sickle cell disease

Sickle cell disease is an inherited condition that causes red blood cells to change shape and block blood vessels. These blockages can lead to severe pain crises, extreme fatigue, and frequent hospital stays. This Phase 3 clinical trial is testing a new medicine called etavopivat to see if it can help children with sickle cell disease. Researchers want to find out whether etavopivat reduces the number of painful crises that require medical attention. They will also check if the medicine improves energy levels, raises healthy blood counts, and reduces time spent in hospital or away from school. Participants will take either etavopivat or a matching placebo (a dummy medicine with no active ingredients). This comparison helps doctors understand if the medicine works well and is safe for young people to take.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Novo Nordisk A/S

Enrolment target

Start

21 Sep 2026

Sickle cell disease

What is this study about?

Sickle cell disease is a lifelong blood condition that affects haemoglobin, the protein in red blood cells that carries oxygen around the body. In people with the condition, red blood cells can become hard and sticky, taking on a crescent or sickle shape. These cells can get stuck in blood vessels, causing sudden episodes of intense pain known as sickle cell crises or vaso-occlusive crises. Over time, these crises can lead to organ damage, severe tiredness, and missed days at school or work.

Etavopivat is an experimental medicine designed to help red blood cells work better. It aims to boost a natural process inside red blood cells so that they hold onto oxygen better, stay round and flexible, and live longer. By keeping red blood cells healthy, researchers hope the medicine will stop blockages from happening in the blood vessels and reduce painful crises.

This Phase 3 trial is an important step in testing whether etavopivat works well and is safe for children. It compares the new medicine against a placebo, which looks identical but contains no active medicine. The results will help doctors see if etavopivat improves day-to-day quality of life, reduces tiredness, and lowers the need for emergency hospital care for young people living with sickle cell disease.

Key takeaways

The study tests whether a new medicine called etavopivat reduces painful sickle cell crises in children.
It is a Phase 3 trial comparing the medicine against a dummy treatment (placebo).
Researchers will track pain crises, tiredness, hospital stays, and missed school days.
Participants will receive regular specialist medical checks and blood monitoring.
Participation is voluntary, and you can leave the study at any time without affecting usual care.

Who may be eligible?

This trial is designed for children and young people diagnosed with sickle cell disease. Both boys and girls can take part. Because this is a paediatric study, parents or legal guardians will need to give permission, and the young person will also be asked to agree if they are old enough.

To join, participants must have a confirmed medical diagnosis of sickle cell disease and generally experience pain crises that have needed medical care in the past. They also need to be able to attend clinic visits and take the study medication as instructed by the hospital team.

Children may not be able to join if they have certain other severe medical conditions, are taking specific medicines that could interfere with the study drug, or have recently had a blood transfusion outside of their usual routine. The study doctors will carry out health checks to make sure taking part is safe for your child.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

My child has a confirmed diagnosis of sickle cell disease.
My child is a child or teenager.
My child has experienced sickle cell pain crises that required medical care.
We can attend regular hospital check-ups and follow the study instructions.
We are willing for my child to take either the study drug or a placebo.

Answer every question to see your result.

What does participation involve?

Taking part in this study involves attending regular appointments at a specialist hospital clinic. Before starting, the study team will carry out health checks, such as physical exams, blood tests, and medical history reviews, to confirm it is safe to join. Once enrolled, participants are randomly chosen by a computer to receive either etavopivat or a matching dummy pill (placebo). Neither you nor your doctor will know which one your child is taking, ensuring the results are fair. Families will be asked to track any painful episodes, record tiredness using simple questionnaires, and note down any missed days of school. Regular hospital check-ups will monitor your child's blood counts, organ health, and general well-being. The study will last for several months to a year or more, and your child's health will be carefully watched throughout.

Potential risks and benefits

Taking part may help improve your child's sickle cell symptoms and could potentially reduce painful crises, though there is no guarantee of direct benefit. Your child will also receive very close medical monitoring by specialist doctors. However, there are potential risks, including unwanted side effects from the study drug, discomfort from blood tests, and the possibility of receiving the placebo instead of the active medicine. Joining the trial is entirely voluntary. You and your child can choose to stop taking part at any time without giving a reason, and this decision will never affect the standard NHS care your child receives.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France
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Unverified
Netherlands
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Unverified
Spain
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Unverified
Italy

Common questions

What is a placebo and why is it used?

A placebo looks and tastes just like the real medicine but contains no active ingredients. It helps researchers prove whether any improvements or side effects are truly caused by the study drug.

Will my child have to stop taking their current sickle cell medicines?

Not necessarily. Many trials allow children to continue certain standard medicines, such as hydroxycarbamide, provided the dose has been stable. The study doctor will check all current medications.

What happens if my child has a severe pain crisis during the trial?

Your child will receive standard emergency medical care straight away. Taking part in the trial will never prevent your child from receiving pain relief or hospital treatment when they need it.

Can we change our minds and leave the study?

Yes. Taking part is completely voluntary. You can withdraw your child from the trial at any point, and their standard NHS treatment will continue as normal.

Will taking part cost our family anything?

The study medicine and tests are provided free of charge. Most clinical trials also help cover reasonable travel expenses and parking costs for clinic visits.