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Autism Spectrum DisorderPhase 2AutismNCT02226458

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

A Phase 2 study testing EPI-743 in people with Autism Spectrum Disorder, run by Edison Pharmaceuticals Inc.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder. Read more about this study

Who is it for?
Ages 3 to 14 years
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
EPI-743
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
31 Oct 2014to 31 Jan 2016
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 14 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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

About this study

Official title: An Exploratory Open-Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder

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

Read the detailed description

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

Being tested
EPI-743
Keywords
Autism, Mitochondrial Disorders, EPI-743, EPI743, Rett syndrome, Angelman syndrome
Medical subject headings
Autism Spectrum Disorder, Autistic Disorder, Mitochondrial Diseases, Rett Syndrome, Angelman Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EPI-743Drug

    Also called Vincerinone TM

Study groups in NCT02226458
Study groupRoleWhat they receive
EPI-743Experimental15 mg/kg oral solution three times per day, maximum of 200 mg per dose
Receives: Drug: EPI-743

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Primary Efficacy

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

    Measured: 6 months

Secondary outcomes (9)

  1. 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

    Measured: 6 months

  2. 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

    Measured: 6 months

  3. 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)

    Measured: 6 months

  4. 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).

    Measured: 6 months

  5. 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)

    Measured: 6 months

  6. Secondary Endpoint

    Change from baseline to six months in intellectual function, attention and memory as assessed by Leiter-R test

    Measured: 6 months

  7. Secondary Endpoint

    Change from baseline to six months in biomarkers of oxidative stress

    Measured: 6 months

  8. 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

    Measured: 6 months

  9. Safety Endpoint

    To examine the safety of EPI-743 in subjects with Autism Spectrum Disorder by examining drug-related adverse and serious adverse events

    Measured: 8 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Aug 2014 (estimated)
  2. Study start31 Oct 2014
  3. Primary completion30 Nov 2015 (estimated)
  4. Study completion31 Jan 2016 (estimated)

Why it stopped: This study was halted prior to enrolling.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02226458
Sponsor's protocol ID
EPI743-14-025
Acronym
Autism
Status last verified
Jan 2022
First submitted
21 Aug 2014
Last updated
21 Jan 2022
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing EPI-743 in people with Autism Spectrum Disorder, run by Edison Pharmaceuticals Inc.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 21 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Primary Efficacy (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02226458.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2022; AzTrials last refreshed its copy on 7 Nov 2026.

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