Phase IIIb Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
Research type
Research Study
Full title
A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants with Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX)
IRAS ID
1006648
Contact name
Maria St. Pierre
Contact email
Sponsor organisation
Amylyx Pharmaceuticals Inc.
Eudract number
2022-002348-33
Research summary
This is an open-label study for participants previously enrolled in Study A35-004 (PHOENIX). All participants will receive open-label treatment with AMX0035 until week 108 or the end of treatment visit. Participants will then be followed up for safety 28 days after the last dose.
A survival follow-up assessment will be completed every 12 weeks following the EOT visit until time of death or EOS.
All participants will be treated with oral (or feeding tube) AMX0035 which comprises of 2 sachets daily (one morning dose and one evening dose) starting on Day 1, for the duration of the study. The study drug is to be mixed with water and taken orally or if applicable, via a feeding tube. If it is noted that twice a day dosing is poorly tolerated, dosing interruptions and reductions may be considered as per protocol
guidance.
The Screening Period is of maximum 6 weeks duration prior to the Baseline Visit (which may be combined with the Week 48 visit of Study A35-004 (PHOENIX).
Overall, the safety and efficacy of edaravone administered by oral and intravenous routes have been demonstrated in many clinical trials in healthy volunteers and ALS patients.
Approximately, 600 patients have been recruited in the main study PHOENIX (21-NREC-CT-022) either randomised to AMX0035 or placebo.
This open-label extension allows previous study participants of the PHOENIX study continued access to AMX0035 for an additional 108 weeks and provides additional evaluation of longitudinal safety and efficacy of treatment with the AMX0035.
The study will also evaluate the effects of the study drug on the participants overall survival, the impact on the participants ALS function and disease progression. Approximately 70 sites in Europe, UK and the US are planned to participate in the
trial.REC name
Wales REC 2
REC reference
22/WA/0365
Date of REC Opinion
22 Aug 2023
REC opinion
Further Information Favourable Opinion