Pozelimab and Cemdisiran for Generalised Myasthenia Gravis
Research type
Research Study
Full title
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis
IRAS ID
304131
Contact name
Saiju Jacob
Contact email
Sponsor organisation
Regeneron Pharmaceuticals, Inc.
Eudract number
2020-003272-41
Clinicaltrials.gov Identifier
147802, IND Number
Duration of Study in the UK
4 years, 6 months, 0 days
Research summary
Myasthenia gravis (MG) is a long-term disease that causes weakness and fatigue in muscles because the nerves and muscles are not communicating properly. It is rare, affecting about 15 in every 100,000 people in the UK. MG mainly affects the muscles that control the eyes, eyelids, facial expressions, chewing, swallowing, speaking and also affects limb muscles. However, it can also affect the muscles between the ribs or diaphragm which impacts breathing and can become life-threatening.
Current treatments include acetylcholinesterase inhibitors with the addition of steroids and immunosuppressants if required. Steroids and immunosuppressants have long-term toxicities and acetylcholinesterase inhibitors produce particular side effects. These treatments also have a long time to onset of effect.
The purpose of this study is to see how effective the combination of pozelimab and cemdisiran is for patients with MG. It is hoped that the combination of these treatments will reduce the levels of complement C5 in the body and improve MG symptoms.
The study includes a 5-week screening period, 24-week double-blind treatment period, 28-week double-blind extension treatment period, 68-week open-label treatment period and a 52-week follow-up period (total duration about 3.5 years).
Patients will be randomized in a 1:1:1 ratio to receive placebo; pozelimab and cemdisiran in combination; or cemdisiran alone. At the end of the double-blind treatment period, placebo patients will be re-randomized in a 1:1 ratio to receive pozelimab and cemdisiran in combination; or cemdisiran alone. They will continue to receive their standard MG treatment throughout the study.
Study treatment is given by injection every 4 weeks. Procedures at study visits include MG questionnaires and assessments, vital signs, physical examinations, ECGs and blood sampling.
This study is sponsored by Regeneron Pharmaceuticals, Inc. Approximately 210 patients will participate in this study worldwide with 2 or more patients from University Hospitals Birmingham.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
21/LO/0751
Date of REC Opinion
17 Dec 2021
REC opinion
Further Information Favourable Opinion