NCT05349721

Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 336 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.

Detailed description

Participants will be randomized to 1 of the 2 treatment groups: PTC857 or matching placebo. Following successful completion of the Treatment Period, participants who enter the LTE Period, will receive open-label PTC857 for 28 weeks. Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	PTC857	PTC8657 will be administered as an oral solution twice a day.
DRUG	Placebo	Matching placebo will be administered as an oral solution twice a day.

Timeline

• Start date: 2022-05-15
• Primary completion: 2024-09-26
• Completion: 2025-01-30
• First posted: 2022-04-27
• Last updated: 2025-08-01
• Results posted: 2025-08-01

Locations

55 sites across 13 countries: United States, Argentina, Australia, Belgium, Czechia, France, Germany, Italy, Japan, Netherlands, Poland, Spain, Sweden

Regulatory

• FDA-regulated drug study