Study Purpose:
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.Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
EPI-589
Placebo:
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Matthew B Klein, MD, FACS, PTC Therapeutics
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
United States
Full Study Summary:
Primary Outcome Measures:
-Effects of EPI-589 on Glutathione in subjects with ALS [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-To evaluate the effects of EPI-589 in Subjects with ALS on blood-based glutathione cycle biomarkers
Secondary Outcome Measures:
-Pharmacokinetics: Drug plasma concentration [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Drug plasma concentration measurements
-Disease progression as assessed by ALS Functional Rating Scale-Revised [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Disease progression as assessed by ALS Functional Rating Scale-Revised
-Respiratory function assessed by PFTs and capnography [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Respiratory function assessed by PFTs and capnography
-Failure to thrive as measured by body weight [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Failure to thrive as measured by body weight
-Effect on swallowing as assessed by change in water and solid swallowing test [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Swallowing as assessed by change in water and solid swallowing test
-Muscle function as assessed by handheld dynamometry [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Muscle function as assessed by handheld dynamometry
-Speech as assessed by speech evaluation [ Time Frame: 6 months ] [ Designated as safety issue: No ]
-Speech as assessed by speech evaluation
Other Outcome Measures:
-Safety as assessed by the number of study drug related adverse events and serious adverse events [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Study Sponsor:
PTC Therapeutics
Participant Duration:
6 months
Estimated Enrollment:
19
Estimated Study Start Date:
01 / 14 / 2016
Estimated Study Completion Date:
02 / 23 / 2018
Posting Last Modified Date:
10 / 14 / 2020
Date Study Added to neals.org:
06 / 02 / 2015
Minimum Age:
21 Years
Maximum Age:
70 Years
Can participants use Riluzole?
Yes
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
Cedar's Sinai
Los Angeles, California
90048
United States
California Pacific Medical Center
San Francisco, California
94115
United States
Providence Brain and Spine Institute ALS Center
Portland, Oregon
97213
United States