Safety and Efficacy Study of Ravulizumab in Adults With gMG
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients with Generalized Myasthenia Gravis
IRAS ID
274001
Contact name
Saiju Jacob
Contact email
Sponsor organisation
Alexion Pharmaceuticals Incorporated
Eudract number
2018-003243-39
Duration of Study in the UK
3 years, 0 months, 18 days
Research summary
Research Summary
This study involves an investigational drug called ravulizumab. This drug is being developed to treat Generalized Myasthenia Gravis (gMG) by blocking complement activity. Complement activity is part of the immune system, which fights against infections. In patients with gMG, abnormal complement activity is present, which causes damage to the structures in the body that are responsible for neuromuscular transmission. Ravulizumab is considered an experimental drug, which means it is not approved for use in gMG in any country at this time. The purpose of this study is to evaluate the safety and efficacy of ravulizumab intravenous (IV; in the vein) in patients with gMG.
Ravulizumab IV has been previously tested in patients with other rare diseases called Paroxysmal Nocturnal Haemoglobinuria (PNH) and atypical Haemolytic Uremic Syndrome (aHUS), which are also caused by abnormal complement activity. However, this study will be the first to test ravulizumab in gMG patients.
It is expected that approximately 140 study sites will be involved around the world, and approximately 160 patients are expected to enrol into the study. The duration of the patient’s participation in the study is anticipated to be approximately 2.5 years, comprising of 4 weeks in the Screening period, 26 weeks in the Randomised-Controlled Treatment Period and 106 weeks in the Open Label Extension (OLE) Period.
During the 4 weeks Screening Period, the patients will undergo different tests and examinations to determine if they qualify to be in the study.
The Randomised-Controlled Treatment Period goes from the first day of study drug administration to Day 183 (26 weeks). This treatment period will consist of 12 study visits.
The Open Label Extension (OLE) Period begins on Day 183 (26 weeks) and will last up to 132 weeks. This treatment period will consist of 18 visits. During the OLE period, patients will receive ravulizumab on Day 183 (Week 26) and will continue for up to 2 years or until the product is registered or approved (in accordance with country-specific regulations), whichever occurs first.
All the examinations and tests conducted during the study are summarised in the Participant Information Form.Summary of Results
A lay summary was published here https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fwww.trialsummaries.com%252FStudy%252FStudyDetails%253Fid%253D25731%2526tenant%253DMT_MED_9011.%2FNBTI%2F-Ui1AQ%2FAQ%2F42e0a2ba-8b2a-415e-9e68-19a2a42a4cb5%2F1%2FupTOaHYnBt&data=05%7C02%7Cedgbaston.rec%40hra.nhs.uk%7C2675c054e93a462f287408dc73222c3f%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638511837803266051%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C0%7C%7C%7C&sdata=zFJMxjnBoRUMF11ClSke9QDd3YAXk6%2BXfwTxL9x3i1o%3D&reserved=0
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0340
Date of REC Opinion
11 Feb 2020
REC opinion
Further Information Favourable Opinion