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

NCT02226458 | Withdrawn Phase 2

• 1 other identifier: EPI743-14-025
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Autism Spectrum Disorder

Keywords

Autism; Mitochondrial Disorders; EPI-743; EPI743; Rett syndrome; Angelman syndrome

Outcome Measures

Primary Outcomes (1)

• Primary Efficacy

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

  6 months

Secondary Outcomes (9)

• Secondary Endpoint

  6 months

• Secondary Endpoint

  6 months

• Secondary Endpoint

  6 months

• Secondary Endpoint

  6 months

• Secondary Endpoint

  6 months

Study Arms (1)

EPI-743

EXPERIMENTAL

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

Drug: EPI-743

Interventions

EPI-743 DRUG

Also known as: Vincerinone TM

EPI-743

Eligibility Criteria

• Age: 3 Years - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Edison Pharmaceuticals Inc: lead

Related Links

• Edison Pharmaceuticals Website

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder; Mitochondrial Diseases; Rett Syndrome; Angelman Syndrome

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Movement Disorders; Central Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Imprinting Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2014
• First Posted: August 27, 2014
• Study Start: October 31, 2014
• Primary Completion: November 30, 2015
• Study Completion: January 31, 2016
• Last Updated: January 21, 2022

Record last verified: 2022-01