Active Lungs & Breathing NIHR-supported project Heart, Stroke & Blood

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children with Hypochondroplasia (Study 111-303)

Funder NIHR (part of DHSC)
Recipient organisation NIHR Great Ormond Street Hospital Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Dec 2027

In plain English

AI plain-English summary

Children with generalised myasthenia gravis (gMG) will receive daily injections of the drug zilucoplan for up to a year, to test its safety and tolerability. gMG is a rare autoimmune disease where the immune system attacks the connection between nerves and muscles, causing muscle weakness, eye problems, and breathing difficulties that can become life-threatening. Few treatments exist for children with gMG, and those that do often cause long-term medical problems. Zilucoplan is designed to block part of the immune attack, offering a potential new treatment option. If the drug proves safe and well tolerated, it could become the first targeted therapy specifically tested for children with this condition. That would give doctors a treatment that avoids the severe side effects of current options, potentially improving long-term health outcomes for a small but vulnerable patient group. The study is an open-label extension, meaning all participants, doctors, and staff know who receives the drug—this design prioritises gathering long-term safety data over blinding.

View original technical description
Researchers are looking for a better way to treat generalised myasthenia gravis (gMG) in children. With gMG, the immune system attacks places where nerve cells connect to muscles. This can lead to muscle weakness, eye problems, and difficulty breathing. Severe cases of gMG can be life-threatening. Few treatments are available for children with gMG. The treatments that do exist can cause long-term medical problems.The study medication, zilucoplan, is designed to stop part of the immune system from attacking the body. Researchers think zilucoplan could be a new treatment option for children with gMG.The aim of the study is to see how safe and well tolerated zilucoplan is when received for over a year.This is a open-label extension study. Open-label means the participants, study doctors, and study staff will know what each participant is receiving. In an extension study, participants from a previous study keep receiving treatment so the researchers can learn more about its safety and how it works. In this extension study, participants will continue to receive treatment with zilucoplan for up to 1 year.Participants will have been 2-17 years of age when the previous study began, will have gMG, must have finished treatment with zilucoplan during their previous study, and the study doctors must think that more treatment with zilucoplan will help them.Zilucoplan is injected by the participant or caregiver through a needle underneath the skin, called a subcutaneous (SC) injection. Doses of zilucoplan are measured in milligrams (mg) per kilogram (kg) of each participant’s body weight. Participants will receive about 0.3 mg/kg of zilucoplan once a day for up to 1 year. Participants will visit the study site every 4-12 weeks while receiving treatment. Zilucoplan injections will happen at home and during study site visits.The study Sponsor is UCB Biopharma SRL.

Researchers

Moira Cheung (Principal Investigator)

Related Research

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A Randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of vosoritide in infants and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery
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