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

NCT02460679 | Completed Phase 2 Results

• 1 other identifier: EPI589-15-001
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Drug-Related Serious Adverse Events (SAEs)

  An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.

  Baseline (Day 0) to Month 6

Secondary Outcomes (19)

• Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12)

  Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3

• Maximum Observed Plasma Concentration (Cmax)

  Pre-dose (0 hour), 0.5, 1, 2, 4, 6, 8, and 12 hours post-dose at Month 1 and 3

• Change From Baseline in ALSFRS-R Total Score at Month 6

  Baseline, Month 6

• Change From Baseline in Vital Capacity at Month 6

  Baseline, Month 6

• Change From Baseline in MIP at Month 6

  Baseline, Month 6

Study Arms (1)

EPI-589

EXPERIMENTAL

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.

Drug: EPI-589

Interventions

EPI-589 DRUG

An immediate release film-coated table at a 250 mg dosage strength will be administered per dose and schedule specified in arm.

Also known as: (R)-troloxamide quinone

EPI-589

Eligibility Criteria

• Age: 21 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (3)

Cedar's Sinai

Los Angeles, California, 90048, United States

Location

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

Providence Brain and Spine Institute ALS Center

Portland, Oregon, 97213, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Patient Advocacy
• Organization: PTC Therapeutics, Inc.

medinfo@ptcbio.com

1-866-562-4620

Study Officials

• Matthew B Klein, MD, FACS

  PTC Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2015
• First Posted: June 2, 2015
• Study Start: January 14, 2016
• Primary Completion: February 23, 2018
• Study Completion: February 23, 2018
• Last Updated: October 14, 2020
• Results First Posted: September 23, 2020

Record last verified: 2020-09

Locations

US (3)