Phase 2 Completed N=19 Treatment

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

Amyotrophic Lateral Sclerosis

Source: ClinicalTrials.gov NCT02460679 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Sep 2020

Primary outcomePrimary: Number of Participants With Drug-Related Serious Adverse Events (SAEs) — 0 Participants

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Drug-Related Serious Adverse Events (SAEs)	—	—
SECONDARY Area Under the Plasma Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12)	7762.7; 6878.8	—
SECONDARY Maximum Observed Plasma Concentration (Cmax)	5179.0; 4167.8	—
SECONDARY Change From Baseline in ALSFRS-R Total Score at Month 6	-4.1	—
SECONDARY Change From Baseline in Vital Capacity at Month 6	-3.1	—
SECONDARY Change From Baseline in MIP at Month 6	2.4	—
SECONDARY Change From Baseline in Respiratory Rate at Month 6	0.3; -0.6; 0.0	—
SECONDARY Change From Baseline in Heart Rate at Month 6	5.2; 7.1; 7.2	—
SECONDARY Change From Baseline in SpO2 at Month 6	-1.0; -0.8; -0.1	—
SECONDARY Change From Baseline in ETCO2 at Month 6	2.384; 0.605; 7.112	—
SECONDARY Failure to Thrive: Number of Participants With Weight Loss of More Than 5 Percent (%) From Baseline at Month 6	2	—
SECONDARY Change From Baseline in Average Solid Swallowing Time and Water Swallowing Time at Month 6	4.80; 6.65	—
SECONDARY Change From Baseline in Muscle Function at Month 6, as Assessed by Handheld Dynamometry Parameters (Grip Strength, Shoulder Flexion, Knee Extension, Hip Flexion, Elbow Flexion, Elbow Extension, and Ankle Dorsi Flexion)	-9.69; -7.28; -4.53; -5.43; -5.42; -5.05	—
SECONDARY Change From Baseline in Number of Words Participant Read at Month 6	0.0	—
SECONDARY Change From Baseline in Time Spent in Reading at Month 6	5.5	—
SECONDARY Change From Baseline in Number of Words Per Minute Read at Month 6	-147.535	—
SECONDARY Number of Participants With Normal Loudness, Normal Nasality, and Normal Intelligibility	10; 6; 6	—
SECONDARY Level of Disease-Related Biomarker (Glutathione) in Plasma	1.34; 2.73	—
SECONDARY Level of Disease-Related Biomarker (Glutathione) in Cerebrospinal Fluid (CSF)	0.13; 0.13	—
SECONDARY Level of Disease-Related Biomarker (Glutathione) in Urine	0.12; 0.17	—

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02460679). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.