RAISE-XT
Research type
Research Study
Full title
A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis
IRAS ID
268475
Contact name
Maria Isabel Leite
Contact email
Sponsor organisation
Ra Pharmaceuticals, Inc.
Eudract number
2019-001565-33
Duration of Study in the UK
2 years, 6 months, 25 days
Research summary
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 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 or immunosuppressionimmunosuppression, which are accompanied by short- and long-term toxicities; or invasive approaches like thymectomy or intravenous immunomodulatory approaches (like IVIg, PLEX).
Most gMG autoantibodies activate a specific immune-pathway, the complement pathway, which damages the AChRs and subsequently muscles fibres. Zilucoplan inhibits the activation of the complement pathway. The purpose of this extension study is to evaluate the long-term safety, efficacy and tolerability profile of zilucoplan in the treatment of generalised MG in participants who have previously taken part in a Ra Pharmaceuticals-sponsored zilucoplan study. Participants will continue to receive zilucoplan until it is approved in their country. In regions where the study is conducted, but zilucoplan is not approved or marketed, it may still be provided to the participants. Approximate enrolment for this study is projected to be 200 participants.
Throughout this extension 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.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
19/SC/0442
Date of REC Opinion
30 Oct 2019
REC opinion
Further Information Favourable Opinion