A Phase 2A Biomarker Study of EPI-589 in Subjects With ALS

Study Purpose:

Open label study with EPI-589 and ALS subjects

Disease:

Amyotrophic Lateral Sclerosis (ALS)

Study Type:

Interventional Trial

Study Category:

Drug Trial, Biomarkers/Imaging

Study Status:

Active, currently recruiting

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Matthew B Klein, MD, FACS Edison Pharmaceuticals

Clinicaltrials.gov ID (11 digit #):

NCT02460679

Neals Affiliated?

No

Coordinating Center Contact Information


Matthew Klein; Erin Johnson / .(JavaScript must be enabled to view this email address) / 650-641-9211; 650-641-9212
.(JavaScript must be enabled to view this email address) 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:

Edison Pharmaceuticals Inc

Participant Duration:

6 months

Estimated Enrollment:

20

Estimated Study Start Date:

11/30/2015

Estimated Study Completion Date:

01/31/2017

Posting Last Modified Date:

06/30/2016

Date Study Added to alsconsortium.org:

07/05/2015
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    70

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    -Dx. of ALS by E1 Escorial Criteria
    -FVC >= 70% years of age
    -Weakness onset within 3 years
    -Agreement to use contraception when age appropriate
    -Willingness and ability to comply with study procedures
    -Stable regimen of dietary supplements and /or riluzole for at least 30 days prior to study
    -Absention from use of other investigative or non-approved drugs
    -Subject must be able to swallow size 0 tablets

    Exclusion Criteria:
    -Allergy to EPI-589
    -Use of ventilation
    -Participation in other intervention studies
    -Inability to undergo MRI or MRS
    -Dx with any other neurologic disease
    -Malignancy within the past 2 years
    -Hx. of stroke
    -Hx. of brain injury
    -Hepatic insufficiency (LFTs > 3 xs ULN)
    -Renal insufficiency requiring dialysis
    -End stage cardiac failure
    -Participation in a trial of a device, drug or other tx. for ALS within 3 months

  • Site Contact Information

    California Pacific Medical Center
    Robert Miller, MD / San Francisco, California 94115
    United States