RAISE
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis
IRAS ID
268190
Contact name
Maria Isabel Leite
Contact email
Sponsor organisation
Ra Pharmaceuticals, Inc.
Eudract number
2019-001564-30
Duration of Study in the UK
0 years, 10 months, 19 days
Research summary
Summary of Research
Myasthenia gravis (MG) is a rare long-term condition characterised by muscle fatigeable weakness, caused by failure of signal transmission from nerve terminals to the muscle fibres. MG is an autoimmune disease, wherein infection-fighting antibodies produced within the body (autoantibodies) wrongly target the signals from the nerves to the muscles. Eye muscles are often the first to be affected, but 85% of cases progress to a more diffuse generalised muscle weakness and fatigue, termed generalised MG (gMG). When respiratory muscles are affected, the symptoms can become life-threatening. In the European Union alone, approximately 191,000 cases of MG are registered.
The most common target of the autoantibodies is the nicotinic Aceylcholine Receptor (AChR), a protein which activates the transmission between the motor neurons and muscle fibres, a physiological process required for the muscles to maintain strength. Current therapies include acetylcholinesterase inhibition (to increase the amount of Aceylcholine avaliable at the junction between the nerve and the muscle) or immunosuppression, which are accompanied by short- and long-term toxicities; or invasive approaches like thymectomy (operation to remove the thymus, to stop production of the autoantibodies) or intravenous immunomodulatory approaches (like IVIg, PLEX).
Most gMG autoantibodies are of a particular type which activate a specific immune-pathway, the complement pathway, which damages the AChRs and subsequently muscle fibres. Zilucoplan acts to inhibit the activation of the complement pathway. The purpose of this clinical study is to evaluate the safety, efficacy and tolerability profile of zilucoplan in the treatment of gMG.
Planned enrolment for this study is 130 participants for a screening period of up to 4 weeks, followed by a treatment period of 12 weeks. Participants will be randomly assigned in a 1:1 ratio to receive either zilucoplan or placebo in a blinded manner.
Throughout the study, participants will be required to complete certain procedures, such as blood tests, urine tests and electrocardiograms. Participants will be recruited at study sites in the UK and globally.
Summary of Results
UCB will be providing plain language summaries of all UCB-sponsored clinical trials conducted in the UK that end in 2022 and beyond. Since this clinical trial ended prior to that date, a plain language summary is not planned at this time.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
19/SC/0441
Date of REC Opinion
30 Oct 2019
REC opinion
Further Information Favourable Opinion