ActiveLungs & BreathingNIHR-supported projectBrain & Nervous System
A Multicentre open-label, uncontrolled study to evaluate the pharmacokinetics, pharmacodynamics, safety, tolerability, and activity of zilucoplan in paediatric study participants from 2 to less than 18 years of age with acetylcholine receptor antibody positive generalised myasthenia gravis.
Recipient organisationNIHR Great Ormond Street Hospital Clinical Research Facility
NIHR supportRecorded as supported by this research centre
PeriodMar 2025 — Nov 2026
In plain English
AI plain-English summary
Children with a rare autoimmune disease that attacks their own muscles are receiving a daily injection of an experimental drug, zilucoplan, to see if it can safely control their symptoms. Generalised myasthenia gravis (gMG) causes muscle weakness, eye problems, and breathing difficulties because the immune system damages the connections between nerves and muscles. Severe cases can be life-threatening. Existing treatments for children are limited and can cause long-term medical problems. Zilucoplan is designed to block a specific part of the immune system from attacking the body, offering a potentially safer alternative. This study will test the drug in at least eight children aged 2 to 17 across several countries. Participants inject 0.3 mg of zilucoplan per kilogram of body weight under the skin once daily for four weeks, with four clinic visits. The total study lasts about 15 weeks, after which children may continue treatment in a follow-up study. If zilucoplan proves safe and effective, it could become the first targeted therapy specifically tested for children with gMG, reducing their reliance on treatments with serious side effects and improving long-term quality of life.
View original technical description
Researchers are looking for a better way to treat generalised myasthenia gravis (gMG) in children. In people with gMG, the immune system attacks the 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. There are few treatments available for children with gMG, and 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 that zilucoplan could be a new treatment option for children with gMG.The aim of the study is to see how safe and effective Zilucoplan is in treating children with positive gMG.This study will include at least 8 participants. All participants will be 2 to 17 years of age and have gMG, across several countries globally. Zilucoplan is injected by the participant or caregiver through a needle just underneath the skin, also called a subcutaneous (SC) injection. Doses of zilucoplan are measured in milligrams (mg) per kilogram (kg) of each participant’s body weight. All participants will receive about 0.3 mg/kg of zilucoplan once a day for 4 weeks. Participants will visit the study site 4 times while they are receiving treatment. Zilucoplan injections will happen at home and during study site visits.Participants will be in this study for up to about 15 weeks. After receiving zilucoplan for 4 weeks in this study, participants may be able to continue receiving zilucoplan in another study. The Sponsor of the study is UCB Biopharma SRL.
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