Phoenix
Research type
Research Study
Full title
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients with Amyotrophic Lateral Sclerosis (ALS)
IRAS ID
299381
Contact name
Christopher McDermott
Contact email
Sponsor organisation
Amylyx Pharmaceuticals Inc.
Eudract number
2021-000250-26
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
2 years, 1 months, 0 days
Research summary
The purpose of this study is to find out if the study drug AMX0035 is safe for the treatment of ALS, and how well it works to determine if AMX0035 can slow down worsening of the disease over 48 weeks compared to placebo in adult patients. Also, AMX0035 is being studied to understand the effect on disease progression in participants with ALS. The sponsor also wants to explore whether the use of AMX0035 influences the burden on the caregiver.
Approximately 600 subjects with ALS and their caregivers are expected to participate. The study takes place in approximately 12 countries (55 sites) total in Europe and in the US.
Participants will first receive an oral dose of the study drug once a day in the morning (AMX0035 or placebo; one sachet per day) for approximately 14-21 days. For participants who tolerate the treatment, the dose will then be escalated to twice a day oral dosing in the morning and evening for the remainder of 48-week treatment duration.
Each participant will be in the trial for approximately 1 year
REC name
Wales REC 2
REC reference
21/WA/0291
Date of REC Opinion
21 Sep 2021
REC opinion
Favourable Opinion