Safety, Tolerability, PK, PD & Activity Study of ZLP in gMG Pediatrics
Research type
Research Study
Full title
AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND ACTIVITY OF ZILUCOPLAN IN PEDIATRIC STUDY PARTICIPANTS WITH ACETYLCHOLINE RECEPTOR ANTIBODY POSITIVE GENERALIZED MYASTHENIA GRAVIS
IRAS ID
1008745
Contact name
Edgar Will
Contact email
Sponsor organisation
UCB Biopharma SRL
Research summary
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.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
23/EM/0233
Date of REC Opinion
15 Nov 2023
REC opinion
Further Information Favourable Opinion