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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date  ICMJE May 21, 2015
First Posted Date  ICMJE June 2, 2015
Last Update Posted Date August 23, 2018
Study Start Date  ICMJE November 2015
Actual Primary Completion Date July 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 14, 2015)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: May 29, 2015)
Effects of EPI-590 on glutathione in subjects with ALS [ Time Frame: 6 months ]
To evaluate the effects of EPI-589 in Subjects with ALS on blood-based glutathione cycle biomarkers
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 14, 2015)
  • Pharmacokinetics: Drug plasma concentration [ Time Frame: 6 months ]
    Drug plasma concentration measurements
  • Disease progression as assessed by ALS Functional Rating Scale-Revised [ Time Frame: 6 months ]
    Disease progression as assessed by ALS Functional Rating Scale-Revised
  • Respiratory function assessed by PFTs and capnography [ Time Frame: 6 months ]
    Respiratory function assessed by PFTs and capnography
  • Failure to thrive as measured by body weight [ Time Frame: 6 months ]
    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 ]
    Swallowing as assessed by change in water and solid swallowing test
  • Muscle function as assessed by handheld dynamometry [ Time Frame: 6 months ]
    Muscle function as assessed by handheld dynamometry
  • Speech as assessed by speech evaluation [ Time Frame: 6 months ]
    Speech as assessed by speech evaluation
  • Changes in biomarkers [ Time Frame: 6 months ]
    Biomarker changes will be measure in blood, cerebrospinal fluid and urine
Original Secondary Outcome Measures  ICMJE
 (submitted: May 29, 2015)
  • Pharmacokinetics: Drug plasma concentration [ Time Frame: 6 months ]
    Drug plasma concentration measurements
  • Disease progression as assessed by ALS Functional Rating Scale-Revised [ Time Frame: 6 months ]
    Disease progression as assessed by ALS Functional Rating Scale-Revised
  • Respiratory function assessed by PFTs and capnography [ Time Frame: 6 months ]
    Respiratory function assessed by PFTs and capnography
  • Failure to thrive as measured by body weight [ Time Frame: 6 months ]
    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 ]
    Swallowing as assessed by change in water and solid swallowing test
  • Muscle function as assessed by handheld dynamometry [ Time Frame: 6 months ]
    Muscle function as assessed by handheld dynamometry
  • Speech as assessed by speech evaluation [ Time Frame: 6 months ]
    Speech as assessed by speech evaluation
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: May 29, 2015)
Safety as assessed by the number of study drug related adverse events and serious adverse events [ Time Frame: 6 months ]
 
Descriptive Information
Brief Title  ICMJE Safety and Biomarker Study of EPI-589 in Subjects With ALS
Official Title  ICMJE A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis
Brief Summary Open label study with EPI-589 and ALS subjects
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE Drug: EPI-589
An immediate release film-coated table at a 250 mg dosage strength.
Other Name: (R)-troloxamide quinone
Study Arms  ICMJE Experimental: EPI-589
EPI-589
Intervention: Drug: EPI-589
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 29, 2015)
20
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 2018
Actual Primary Completion Date July 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 21 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02460679
Other Study ID Numbers  ICMJE EPI589-15-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Edison Pharmaceuticals Inc
Study Sponsor  ICMJE Edison Pharmaceuticals Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Matthew B Klein, MD, FACS Edison Pharmaceuticals
PRS Account Edison Pharmaceuticals Inc
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP