A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of SAR443820 in Adult Participants With Amyotrophic Lateral Sclerosis, Followed by an Open-label Extension (HIMALAYA)
Study Purpose:
This is a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants,18 to 80 years of age with ALS followed by an openlabel, longterm extension period.
Study ACT16970 consists of 2 parts (A and B) as follows:
Part A is a 24 week, double blind, placebo controlled part, preceded by a screening period of up to 4 weeks before Day 1.
On Day 1 of Part A, participants will be randomized in a 2:1 ratio to the SAR443820 treatment arm or matching placebo arm as listed below:
Treatment arm: SAR443820, BID
Placebo arm: Placebo, BID
Randomization will be stratified by the geographic region of the study site, region of ALS onset (bulbar vs other areas), use of riluzole (yes vs no), and use of edaravone (yes vs no). Participants will attend inclinic study assessments at baseline (Day 1), Week 2, Week 4, Week 8, Week 16, and Week 24, and will receive a phone call at Week 12 and Week 20. After successful completion of Part A, all participants will rollover to Part B. The Week 24 Visit is the end of Part A and the beginning of Part B.
Part B is an open label, longterm extension period that starts from the end of Part A (Week 24) and continues up to Week 106. The objectives of Part B are to further determine the safety and efficacy of longterm SAR443820 treatment. The treatment assignment of participants in Part A will remain blinded to Investigators, participants, and site personnel until the end of Part B. Every participant will receive BID oral tablets of SAR443820 in Part B.
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
EnrollingPhase:
Phase IIStudy Chair(s)/Principal Investigator(s):
Clinicaltrials.gov ID (11 digit #):
NCT05237284Neals Affiliated?
NoCoordinating Center Contact Information
Sanofi.(JavaScript must be enabled to view this email address) / 800-633-1610 ext option 6
.(JavaScript must be enabled to view this email address)
Full Study Summary:
Part A of the study will last for 24 weeks, and participants will receive BID oral SAR443820 or placebo in a doubleblind fashion for 24 weeks. Participants who successfully complete double blind Part A will rollover to open label Part B.
Part B begins at the end of Week 24 and continues up to Week 106. All participants will receive BID oral tablets of SAR443820 in Part B.
The study duration includes an up to 4-week screening period, 24-week double blind treatment period in Part A, 80-week open label treatment period in Part B, and 2-week post treatment follow up period, with a maximum total study duration of 110 weeks.