NCT05619783

Extension Study Evaluating The Safety And Tolerability of AMX0035

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)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 352 (actual)
• Sponsor: Amylyx Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective is to evaluate the safety and tolerability of AMX0035 over 108 weeks of open label treatment for participants previously enrolled in Study A35-004 (PHOENIX).

Detailed description

All participants will receive open-label treatment with AMX0035, starting on Day 1 with twice a day oral dosing (once in the morning and once in the evening) for the duration of the study. After the Baseline Visit (Day 1), enrolled participants will complete visits approximately every 12 weeks (± 2 weeks), until Week 108 or the end of treatment (EOT) visit, followed by a safety follow-up approximately 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 end of study (EOS).

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	AMX0035	Combination of 3 g phenylbutyrate and 1 g taurursodiol

Timeline

• Start date: 2022-12-29
• Primary completion: 2024-10-16
• Completion: 2024-10-30
• First posted: 2022-11-17
• Last updated: 2025-01-13

Locations

46 sites across 11 countries: Belgium, France, Germany, Ireland, Italy, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study