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

Edison Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Autism Spectrum Disorder

Treatments: Drug: EPI-743

Study type: Interventional

Funder types: Industry

Identifiers

NCT02226458

EPI743-14-025

Details and patient eligibility

About

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

Full description

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

Sex: All

Ages: 3 to 14 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

EPI-743

Experimental group

Description:

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

Treatment:

Drug: EPI-743