ALXN1210-ALS-308_Ravulizumab in patients with ALS
Research type
Research Study
Full title
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS)
IRAS ID
278838
Contact name
Pamela Shaw
Contact email
Sponsor organisation
Alexion Pharmaceuticals
Eudract number
2019-004619-30
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
145660, IND Number
Duration of Study in the UK
4 years, 2 months, 31 days
Research summary
Amyotrophic Lateral Sclerosis (ALS) is a condition that affects parts of the nervous system that control voluntary muscle movements. Voluntary muscles produce movements like chewing, talking, swallowing, breathing, moving the arms and hands and walking. The disease is progressive, meaning the symptoms get worse over time leading to severe disability.
This study involves research of an investigational drug called ravulizumab. This drug is being developed to treat ALS by blocking complement activity. The complement system is part of the immune system which fights against infections. In patients with ALS, abnormal complement activity is present, which may cause damage to the structures in the body that are responsible for neuromuscular transmission.
Ravulizumab is an experimental drug, not approved for ALS. About 90 study sites will be opened around the world to enroll 354 participants with ALS. In the UK it is expected that the study will take place in several sites.
Ravulizumab has been previously tested in patients with other rare diseases which are also related to by abnormal complement activity. Ravulizumab has been approved under the brand name ULTOMIRIS®™ to treat Paroxysmal Nocturnal Hemoglobinuria (PNH – a rare blood condition) in the US, Europe, and Japan, and to treat atypical Hemolytic Uremic Syndrome (aHUS – a rare disease that can lead to kidney failure) in the US. However, this study will be the first to test ravulizumab in participants with ALS.
The study comprises of a Screening Period (up to 4 weeks), a Randomised Controlled Period (50 weeks), and an Open-Label Extension Period. The Open-Label Extension Period will continue for up to 2 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.
Eligible participants will be randomised (2:1) to receive intravenous (IV) infusion of ravulizumab or matching placebo until Week 50 during the double-blind Randomised Controlled Period. All participants will receive ravulizumab treatment during the Open-Label Extension Period.
After the end of treatment visit or early discontinuation, participants will be followed for an additional 8 weeks after their last dose of study drug.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
20/YH/0102
Date of REC Opinion
26 May 2020
REC opinion
Further Information Favourable Opinion