Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)

• Status: Completed
• Trial end date: 2018-02-23
• Target enrollment: 0
• Participant gender: All

Summary

This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of EPI-589 therapy on known trajectory.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edison Pharmaceuticals Inc; PTC Therapeutics

Criteria

Inclusion Criteria:

• Diagnosis of possible, probable, laboratory supported probable, or definite ALS by E1 Escorial Criteria

• Forced vital capacity (FVC) ≥ 70% of predicted

• Weakness onset within 3 years

• Agreement to use contraception if within reproductive years

• Willingness and ability to comply with study procedures

• Stable regimen of dietary supplements and /or riluzole for at least 30 days prior to enrollment

• Abstention from use of other investigative or non-approved drugs

• Participants must be able to swallow 0.375 * 0.700 inch tablets

Exclusion Criteria:

• Allergy to EPI-589

• Use of ventilation

• Participation in other intervention studies

• Diagnosis of any other neurologic disease

• Malignancy within the past 2 years

• History of stroke

• History of brain surgery

• Hepatic insufficiency with liver function tests (LFTs) greater than 3 times upper limit of normal (ULN)

• Renal insufficiency requiring dialysis

• End stage cardiac failure

• Participation in a trial of a device, drug, or other therapy for ALS within 3 months of screening or during the trial