MERIDIAN
Research type
Research Study
Full title
A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in subjects with Amyotrophic Lateral Sclerosis (ALS)
IRAS ID
272854
Contact name
Ammar Al-Chalabi
Contact email
Sponsor organisation
Apellis Pharmaceuticals, Inc.
Eudract number
2019-003797-10
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
2 years, 6 months, 1 days
Research summary
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurological disease that affects nerves in the brain and spinal cord, subsequently causing loss of muscle control, and ultimately death. It may initially present as muscle weakness in the limbs or difficulty in swallowing or breathing. About 10% of cases are hereditary while the cause for about 20% of the remaining cases is genetic, despite a lack of family history.
There is no known cure for ALS. Riluzole and edavarone, the two approved medications, are not adequate to manage ALS in a meaningful way in the majority of the patient population. Consequently, there exists a large unmet need for an effective strategy to manage ALS.
This study will evaluate the efficacy and safety of pegcetacoplan in approximately 228 patients with ALS over 116 weeks. Clinical studies indicate that pegcetacoplan has the potential to delay disease progression by targeting the C3 protein of the complement system, which is a part of the immune system that enhances the ability of the body to get rid of microbes and damaged cells.
After a 6-week screening period, patients will be randomly assigned to receive a bi-weekly dose of 1080 mg of pegcetacoplan or placebo for 52 weeks, after which all patients will receive pegcetacoplan at the same dose for a further 52 weeks. The off-treatment follow-up period is 6 weeks.
Patients will undergo physical examinations, electrocardiograms, blood and urine tests, and will have to complete questionnaires to measure disease progress.REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
21/NE/0067
Date of REC Opinion
28 Apr 2021
REC opinion
Further Information Favourable Opinion