AuthorisedTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Transfusion-Dependent Thalassemia (ENERGIZEKids-T)

Thalassaemia is an inherited blood condition where the body does not make enough healthy red blood cells. Children with severe thalassaemia rely on regular blood transfusions every few weeks to stay well. While vital, these transfusions can be time-consuming and lead to iron building up in the body. This study, called ENERGIZEKids-T, is testing a new tablet called mitapivat in children and young people with alpha- or beta-thalassaemia. Mitapivat is designed to give red blood cells more energy, helping them survive longer. Participants will receive either mitapivat or a dummy pill (placebo) for 48 weeks. Neither the families nor the doctors will know who gets which treatment during the study. Doctors will track whether the medicine cuts down the need for blood transfusions by at least 50%, while keeping a close eye on general growth, hormone levels, and safety.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Agios Pharmaceuticals Inc.

Enrolment target

Start

20 Aug 2026

Transfusion-Dependent Alpha- or Beta-Thalassemia

What is this study about?

Thalassaemia is an inherited condition affecting haemoglobin, the protein in red blood cells that carries oxygen around the body. People with transfusion-dependent thalassaemia cannot produce enough healthy red blood cells on their own. Children with this condition need regular blood transfusions throughout their lives to stay active and healthy. However, having blood transfusions every few weeks means spending many hours in hospital, and over time, repeated transfusions can lead to extra iron collecting in the organs, which requires separate daily treatments to manage.

Researchers are looking for new treatments that can help the body's red blood cells work better. Mitapivat is a medicine that boosts a natural enzyme inside red blood cells, giving them the energy they need to stay healthy and last longer in the bloodstream. If it works as hoped, it could mean that children need fewer transfusions of donor blood.

This trial aims to confirm whether mitapivat is safe and effective for children and teenagers with either alpha- or beta-thalassaemia. The main goal is to see if patients taking mitapivat can reduce their need for blood transfusions by 50% or more over a 12-week period during the 48-week study. Researchers will also check whether some young people can go for periods without any transfusions at all, and whether the treatment helps lower unwanted iron build-up in the body.

Key takeaways

Tests a new daily tablet called mitapivat for children and teens with transfusion-dependent thalassaemia.
Aims to reduce the amount of blood transfusions needed by 50% or more.
A randomised study where participants receive either mitapivat or a dummy tablet for 48 weeks.
Participants will still receive blood transfusions whenever their healthcare team says they need one.
Taking part is voluntary, and you can leave the study at any point without changing your normal care.

Who may be eligible?

This study is designed for children and young people who have been diagnosed with transfusion-dependent thalassaemia (either alpha- or beta-type). To take part, participants must need regular, scheduled blood transfusions to keep their blood counts steady, with good medical records showing their transfusion history over recent months.

Both boys and girls can participate. Before joining, the study doctor will carry out general health checks, including blood tests, to make sure it is safe for the child to enter the study. The child and their parents or guardians must also be willing and able to attend planned clinic visits and follow the study instructions.

Children may not be able to join if they have certain other serious medical conditions, severe liver or kidney problems, or if they have recently received specific new medications or gene therapies. The study doctor will review each child's full medical history to decide if the trial is a safe match.

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 is a child or teenager diagnosed with alpha- or beta-thalassaemia.
My child currently needs regular, routine blood transfusions to stay healthy.
We have a reliable record of my child's transfusion history over the past year.
My child can swallow tablets or take oral medicine regularly.
We are able to travel to a trial centre for regular check-ups and monitoring visits.

Answer every question to see your result.

What does participation involve?

Taking part in this study lasts around one year (48 weeks of treatment) plus follow-up visits. Participants are randomly put into one of two groups: two out of three will receive mitapivat, and one out of three will receive a matching dummy pill (placebo). The medicine is taken by mouth as a tablet. Throughout the study, participants continue to receive their standard medical care and will still get blood transfusions whenever their clinical team decides they need them. Families will visit the study clinic regularly for check-ups, which include blood and urine tests, physical examinations, and questions about how the child is feeling. Doctors will also carefully track the child's height, weight, natural growth development, and iron levels. After the main study ends, there may be an option to continue receiving the medicine in an extension study.

Potential risks and benefits

Taking part might help reduce how much donor blood your child needs, which could mean fewer hospital trips and lower iron levels in the body. The study also helps doctors learn more about better ways to treat thalassaemia in young people. However, there is no guarantee of direct benefit, and your child might receive the dummy pill instead of the active medicine. There may also be side effects from mitapivat, such as headaches, nausea, or changes in hormone levels, and having regular blood tests can be uncomfortable. You and your child can choose to leave the study at any time without giving a reason, and your regular NHS medical care will not be affected in any way.

Locations (2)

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

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Unverified
Italy
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Unverified
Greece

Common questions

What is a placebo and why is it used?

A placebo is a dummy pill that looks and tastes exactly like the real medicine but contains no active ingredients. Researchers use it to see whether changes in health are truly caused by the study medicine rather than by chance.

Will my child still get blood transfusions during the trial?

Yes. Your child's safety always comes first. They will continue to be monitored by their doctors and will receive blood transfusions whenever their blood levels show they need one.

How is the study medicine taken?

Mitapivat (or the matching placebo) is taken by mouth as a tablet, usually once or twice a day with water, as directed by the study team.

Can we drop out if we change our minds?

Yes. Joining the trial is completely voluntary. You or your child can decide to stop taking part at any point, and your usual healthcare team will continue to support you as normal.

Does taking part cost anything?

No. The study medication, tests, and doctor visits related to the trial are provided free of charge, and help with travel expenses to the hospital is usually available.