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An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder (Autism)

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ClinicalTrials.gov Identifier: NCT02226458
Recruitment Status : Withdrawn (This study was halted prior to enrolling.)
First Posted : August 27, 2014
Last Update Posted : March 10, 2015
Sponsor:
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Brief Summary:
The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: EPI-743 Phase 2

Detailed Description:
An Exploratory Open Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children with Autism Spectrum Disorder

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Exploratory Open-Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
Study Start Date : October 2014
Estimated Primary Completion Date : November 2015
Estimated Study Completion Date : January 2016


Arm Intervention/treatment
Experimental: EPI-743
15 mg/kg oral solution three times per day, maximum of 200 mg per dose
Drug: EPI-743
Other Name: Vincerinone TM




Primary Outcome Measures :
  1. Primary Efficacy [ Time Frame: 6 months ]
    Change in plasma levels of reduced and oxidized glutathione from baseline to six months


Secondary Outcome Measures :
  1. Secondary Endpoint [ Time Frame: 6 months ]
    Change in language skills as assessed by change from baseline to six months on the Clinical Evaluation of Language Fundamentals or Preschool Language Scale

  2. Secondary Endpoint [ Time Frame: 6 months ]
    Change from baseline to six months in adaptive behavior and social skills as assessed by the Vineland Adaptive Behavior Scale-Second Edition (VABS), Social Responsiveness Scale (SRS), and computerized eye movement analysis

  3. Secondary Endpoint [ Time Frame: 6 months ]
    Change from baseline to six months in stereotyped behavior and associated ASD symptoms as assessed by Aberrant Behavior Checklist (ABC), The Ohio Autism Clinical Impression Scale (OACIS), Repetitive Behavior Scale - Revised (RBS-R) and Autism symptoms questionnaire (ASQ)

  4. Secondary Endpoint [ Time Frame: 6 months ]
    Change from baseline to six months in gastrointestinal function as assessed by Symptom Diary (stool frequency, abdominal symptoms and medications) and six-item GI severity index (6-GSI).

  5. Secondary Endpoint [ Time Frame: 6 months ]
    Change from baseline to six months in caregiver strain experienced by parents of children with emotional and behavioral disorders as assessed by Caregiver Strain Questionnaire (CGSQ)

  6. Secondary Endpoint [ Time Frame: 6 months ]
    Change from baseline to six months in intellectual function, attention and memory as assessed by Leiter-R test

  7. Secondary Endpoint [ Time Frame: 6 months ]
    Change from baseline to six months in biomarkers of oxidative stress

  8. Secondary Endpoint [ Time Frame: 6 months ]
    Pharmacokinetic Assessment of EPI-743 including maximal plasma concentration (Cmax), area under plasma concentration curve (AUC), apparent oral clearance, apparent volume of distribution and time to peak concentration

  9. Safety Endpoint [ Time Frame: 8 months ]
    To examine the safety of EPI-743 in subjects with Autism Spectrum Disorder by examining drug-related adverse and serious adverse events



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Ages Eligible for Study:   3 Years to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of Autism Spectrum Disorder as defined by the DSM-V criteria for ASD and a gold-standard diagnostic evaluation for ASD such as the Autism Diagnostic Observation Schedule (ADOS) and/or the Autism Diagnostic Interview - Revised (ADI-R)
  2. Male or female, 3 years to 14 years of age
  3. Abnormal glutathione cycle biomarkers (GSH/GSSG in plasma<8.0)
  4. Language impairment (as defined by the CELF-2 or CELF-4 screener)
  5. Ability to complete language assessment (using either CELF or PLS)
  6. Subject or subject's guardian able to consent and comply with protocol requirements
  7. Abstention from use of Coenzyme Q10, vitamin E, lipoic acid, folinic acid, other forms of folic acid above recommended daily allowance (RDA), and idebenone 2 months prior to treatment with EPI-743 and for duration of study
  8. Stable regimen of medication and supplements for 2 months prior to enrollment and 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. Use of anticoagulant medications
  5. Participation in any other interventional study within 90 days of treatment.
  6. Use of antipsychotic medications
  7. Moderate to severe positive response on ABC irritability subscale on questions: Injures self on purpose, is aggressive to other children or adults (verbally or physically), deliberately hurts himself/ herself, and/or does physical violence to self
  8. Severe impairment as defined as a Vineland Adaptive Behavioral Scales composite standard score of <40
  9. Patients with genetic disease that gives rise to ASD (e.g. Rett syndrome, Down syndrome, fragile x syndrome)
  10. Abnormal liver function tests two times the upper limit of normal or renal insufficiency with creatinine levels two times the upper limit of normal

Responsible Party: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT02226458     History of Changes
Other Study ID Numbers: EPI743-14-025
First Posted: August 27, 2014    Key Record Dates
Last Update Posted: March 10, 2015
Last Verified: March 2015

Keywords provided by Edison Pharmaceuticals Inc:
Autism
Mitochondrial Disorders
EPI-743
EPI743
Rett syndrome
Angelman syndrome

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders
Ubiquinone
Micronutrients
Growth Substances
Physiological Effects of Drugs