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

Phase 2

Withdrawn

• Conditions: Autism Spectrum Disorder
• Interventions: Drug: EPI-743

• Registration Number: NCT02226458
• Lead Sponsor: Edison Pharmaceuticals Inc

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

Study Timeline

• Study First Submitted: 2014-08-21
• Study First Submitted QC: 2014-08-25
• Study First Posted: 2014-08-27
• Study Start: 2014-10-31
• Primary Completion: 2015-11-30
• Study Completion: 2016-01-31
• Status Verified: 2022-01
• Last Update Submitted: 2022-01-20
• Last Update Posted: 2022-01-21

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
EPI-743	EPI-743	15 mg/kg oral solution three times per day, maximum of 200 mg per dose

Primary Outcome Measures

Name	Time	Method
Primary Efficacy	6 months	Change in plasma levels of reduced and oxidized glutathione from baseline to six months

Secondary Outcome Measures

Name	Time	Method
Secondary Endpoint	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
Safety Endpoint	8 months	To examine the safety of EPI-743 in subjects with Autism Spectrum Disorder by examining drug-related adverse and serious adverse events