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Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

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. Identifier: NCT02352896
Recruitment Status : Active, not recruiting
First Posted : February 2, 2015
Last Update Posted : July 17, 2019
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Brief Summary:
EPI-743 in Leigh syndrome subjects that participated in previous EPI743-12-002 study.

Condition or disease Intervention/treatment Phase
Leigh Syndrome Drug: EPI-743 Phase 2

Detailed Description:
To monitor the long-term safety and neurodevelopmental effects of EPI-743 on children who complete the EPI743-12-002 placebo controlled trial.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 31 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: EPI743-13-023: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Study Start Date : January 2014
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: EPI-743
EPI-743 administered at a dose of 15 mg/kg up to a total 200 mg three times daily
Drug: EPI-743
EPI-743 is a quinone oxidation product of alpha tocotrienol
Other Name: VincerinoneTM

Primary Outcome Measures :
  1. Long term effect of EPI743 on disease severity as measured by NPMDS [ Time Frame: Up to 3 years ]

Secondary Outcome Measures :
  1. Effect of EPI743 on neurodevelopment as measured by Bayley or Wechsler Scales [ Time Frame: 2 years ]

  2. Effect of EPI743 on neuromuscular function as assesed by Barry Albright Dystonia [ Time Frame: 2 years ]
  3. Effect of EPI743 on Respiratory Function as measured by oxygen saturation [ Time Frame: Up to 3 years ]
  4. Effect of EPI743 on disease morbidity and mortality as measure by mortality, medical encounters and hospitallizations [ Time Frame: Up to 3 years ]
  5. Effect of EPI743 on Safety as measure by differences in adverse events [ Time Frame: Up to 3 years ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Diagnosis of Leigh syndrome with genetic confirmation
  2. Completion of EPI743-12-002 protocol and initiation of treatment within 14 days of Month 12 Visit in EPI743-12-002 study
  3. Patient or Patient's guardian able to consent and comply with protocol requirements
  4. Continued abstention from supplements excluded in EPI743-12-002 study
  5. Continued abstention from Botox® and for the duration of the study

Exclusion Criteria:

  1. Allergy to EPI-743 or sesame oil
  2. Allergy to vitamin E
  3. Clinical history of bleeding or abnormal baseline PT/PTT
  4. Hepatic insufficiency with LFTs greater than two times upper limit of normal
  5. Renal insufficiency requiring dialysis
  6. End stage cardiac failure
  7. Fat malabsorption syndromes precluding drug absorption
  8. Use of anticoagulant medications
  9. Participation in other clinical research studies/taking other experimental agents
  10. Participation in elective procedures that required sedation

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 identifier (NCT number): NCT02352896

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United States, California
Gregory Enns
Stanford University, California, United States, 94305
United States, Ohio
Akron Children's Hospital
Akron, Ohio, United States, 44308
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Edison Pharmaceuticals Inc
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Principal Investigator: Bruce Cohen, MD Akron Children's Hospital
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Responsible Party: Edison Pharmaceuticals Inc Identifier: NCT02352896    
Other Study ID Numbers: EPI743-13-023
First Posted: February 2, 2015    Key Record Dates
Last Update Posted: July 17, 2019
Last Verified: July 2019
Keywords provided by Edison Pharmaceuticals Inc:
Leigh syndrome
Mitochondrial disorders
Additional relevant MeSH terms:
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Leigh Disease
Pathologic Processes
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Pyruvate Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors
Metabolic Diseases
Mitochondrial Diseases