Clinical trial NCT02226458: An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
Official title: An Exploratory Open-Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: Autism
Mitochondrial Disorders
EPI-743
EPI743
Rett syndrome
Angelman syndrome
Lead sponsor: Edison Pharmaceuticals Inc
Sponsor class: INDUSTRY
First posted: 2014-08-27
Enrollment: 0
Sex: ALL
Minimum age (years): 3
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Eligibility 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

Primary outcomes:
measure: Primary Efficacy
timeFrame: 6 months
Secondary outcomes:
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Secondary Endpoint
timeFrame: 6 months
measure: Safety Endpoint
timeFrame: 8 months
Other outcomes:
Locations:
Study officials:
References: