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Safety and Biomarker Study of EPI-589 in Subjects With ALS (EPI-589)

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ClinicalTrials.gov Identifier: NCT02460679
Recruitment Status : Completed
First Posted : June 2, 2015
Last Update Posted : August 23, 2018
Sponsor:
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Brief Summary:
Open label study with EPI-589 and ALS subjects

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: EPI-589 Phase 2

Detailed Description:
Open label with 30-day run in phase to establish baseline parameters and a 90-day withdrawal phase to determine duration of treatment response.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis
Study Start Date : November 2015
Actual Primary Completion Date : July 2018
Actual Study Completion Date : July 2018


Arm Intervention/treatment
Experimental: EPI-589
EPI-589
Drug: EPI-589
An immediate release film-coated table at a 250 mg dosage strength.
Other Name: (R)-troloxamide quinone




Primary Outcome Measures :
  1. Effects of EPI-589 on safety as assessed by drug-related AEs [ Time Frame: 6 months ]
    To evaluate the effects of EPI-589 in Subjects with ALS on drug related adverse events.


Secondary Outcome Measures :
  1. Pharmacokinetics: Drug plasma concentration [ Time Frame: 6 months ]
    Drug plasma concentration measurements

  2. Disease progression as assessed by ALS Functional Rating Scale-Revised [ Time Frame: 6 months ]
    Disease progression as assessed by ALS Functional Rating Scale-Revised

  3. Respiratory function assessed by PFTs and capnography [ Time Frame: 6 months ]
    Respiratory function assessed by PFTs and capnography

  4. Failure to thrive as measured by body weight [ Time Frame: 6 months ]
    Failure to thrive as measured by body weight

  5. Effect on swallowing as assessed by change in water and solid swallowing test [ Time Frame: 6 months ]
    Swallowing as assessed by change in water and solid swallowing test

  6. Muscle function as assessed by handheld dynamometry [ Time Frame: 6 months ]
    Muscle function as assessed by handheld dynamometry

  7. Speech as assessed by speech evaluation [ Time Frame: 6 months ]
    Speech as assessed by speech evaluation

  8. Changes in biomarkers [ Time Frame: 6 months ]
    Biomarker changes will be measure in blood, cerebrospinal fluid and urine



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Ages Eligible for Study:   21 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02460679


Locations
United States, California
Cedar's Sinai
Los Angeles, California, United States, 90048
California Pacific Medical Center
San Francisco, California, United States, 94115
United States, Oregon
Providence Brain and Spine Institute ALS Center
Portland, Oregon, United States, 97213
Sponsors and Collaborators
Edison Pharmaceuticals Inc
Investigators
Study Director: Matthew B Klein, MD, FACS Edison Pharmaceuticals

Responsible Party: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT02460679     History of Changes
Other Study ID Numbers: EPI589-15-001
First Posted: June 2, 2015    Key Record Dates
Last Update Posted: August 23, 2018
Last Verified: August 2018

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases