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

PTC Therapeutics

Status and phase: Completed; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: EPI-589

Study type: Interventional

Funder types: Industry

Identifiers

NCT02460679

EPI589-15-001

Details and patient eligibility

About

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.

Enrollment: 19 patients

Sex: All

Ages: 21 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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19 participants in 1 patient group

EPI-589

Experimental group

Description:

Participants will receive EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues.

Treatment:

Drug: EPI-589