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

Primary Purpose

Autism Spectrum Disorder

Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
EPI-743
Sponsored by
Edison Pharmaceuticals Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Autism, Mitochondrial Disorders, EPI-743, EPI743, Rett syndrome, Angelman syndrome

Eligibility Criteria

3 Years - 14 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    EPI-743

    Arm Description

    15 mg/kg oral solution three times per day, maximum of 200 mg per dose

    Outcomes

    Primary Outcome Measures

    Primary Efficacy
    Change in plasma levels of reduced and oxidized glutathione from baseline to six months

    Secondary Outcome Measures

    Secondary Endpoint
    Change in language skills as assessed by change from baseline to six months on the Clinical Evaluation of Language Fundamentals or Preschool Language Scale
    Secondary Endpoint
    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
    Secondary Endpoint
    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)
    Secondary Endpoint
    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).
    Secondary Endpoint
    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)
    Secondary Endpoint
    Change from baseline to six months in intellectual function, attention and memory as assessed by Leiter-R test
    Secondary Endpoint
    Change from baseline to six months in biomarkers of oxidative stress
    Secondary Endpoint
    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
    Safety Endpoint
    To examine the safety of EPI-743 in subjects with Autism Spectrum Disorder by examining drug-related adverse and serious adverse events

    Full Information

    First Posted
    August 21, 2014
    Last Updated
    January 20, 2022
    Sponsor
    Edison Pharmaceuticals Inc
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02226458
    Brief Title
    An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
    Acronym
    Autism
    Official Title
    An Exploratory Open-Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    January 2022
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    This study was halted prior to enrolling.
    Study Start Date
    October 31, 2014 (Actual)
    Primary Completion Date
    November 30, 2015 (Anticipated)
    Study Completion Date
    January 31, 2016 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Edison Pharmaceuticals Inc

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.
    Detailed Description
    An Exploratory Open Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children with Autism Spectrum Disorder

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Autism Spectrum Disorder
    Keywords
    Autism, Mitochondrial Disorders, EPI-743, EPI743, Rett syndrome, Angelman syndrome

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    EPI-743
    Arm Type
    Experimental
    Arm Description
    15 mg/kg oral solution three times per day, maximum of 200 mg per dose
    Intervention Type
    Drug
    Intervention Name(s)
    EPI-743
    Other Intervention Name(s)
    Vincerinone TM
    Primary Outcome Measure Information:
    Title
    Primary Efficacy
    Description
    Change in plasma levels of reduced and oxidized glutathione from baseline to six months
    Time Frame
    6 months
    Secondary Outcome Measure Information:
    Title
    Secondary Endpoint
    Description
    Change in language skills as assessed by change from baseline to six months on the Clinical Evaluation of Language Fundamentals or Preschool Language Scale
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    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
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    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)
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    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).
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    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)
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    Change from baseline to six months in intellectual function, attention and memory as assessed by Leiter-R test
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    Change from baseline to six months in biomarkers of oxidative stress
    Time Frame
    6 months
    Title
    Secondary Endpoint
    Description
    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
    Time Frame
    6 months
    Title
    Safety Endpoint
    Description
    To examine the safety of EPI-743 in subjects with Autism Spectrum Disorder by examining drug-related adverse and serious adverse events
    Time Frame
    8 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    3 Years
    Maximum Age & Unit of Time
    14 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: 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) Male or female, 3 years to 14 years of age Abnormal glutathione cycle biomarkers (GSH/GSSG in plasma<8.0) Language impairment (as defined by the CELF-2 or CELF-4 screener) Ability to complete language assessment (using either CELF or PLS) Subject or subject's guardian able to consent and comply with protocol requirements 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 Stable regimen of medication and supplements for 2 months prior to enrollment and duration of the study Exclusion Criteria: Allergy to EPI-743 or sesame oil Allergy to vitamin E Clinical history of bleeding or abnormal baseline PT/PTT Use of anticoagulant medications Participation in any other interventional study within 90 days of treatment. Use of antipsychotic medications 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 Severe impairment as defined as a Vineland Adaptive Behavioral Scales composite standard score of <40 Patients with genetic disease that gives rise to ASD (e.g. Rett syndrome, Down syndrome, fragile x syndrome) Abnormal liver function tests two times the upper limit of normal or renal insufficiency with creatinine levels two times the upper limit of normal

    12. IPD Sharing Statement

    Links:
    URL
    http://edisonpharma.com/
    Description
    Edison Pharmaceuticals Website

    Learn more about this trial

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

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