An Exploratory Open Label Study of EPI-743 (Vincerinone T... | NCT02226458

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

NCT02226458

Sponsor

Edison Pharmaceuticals Inc

Status

Withdrawn

Last Update Posted

Jan 21, 2022Actual

Enrollment

0Actual

Phase

Phase 2

Conditions

Autism Spectrum Disorder

Interventions

EPI-743

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jan 2022

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

This study was halted prior to enrolling.

Expanded Access Info

Start Date

Oct 31, 2014Actual

Primary Completion Date

Nov 30, 2015Estimated

Completion Date

Jan 31, 2016Estimated

First Submitted Date

Aug 21, 2014

First Submission Date that Met QC Criteria

Aug 25, 2014

First Posted Date

Aug 27, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 20, 2022

Last Update Posted Date

Jan 21, 2022Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Label	Type	Description	Intervention Names
EPI-743	Experimental	15 mg/kg oral solution three times per day, maximum of 200 mg per dose	Drug: EPI-743

Name	Type	Description	Arm Group Labels	Other Names
EPI-743	Drug		EPI-743	Vincerinone TM

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Primary Efficacy	Change in plasma levels of reduced and oxidized glutathione from baseline to six months	6 months

Secondary Outcomes

Measure	Description	Time Frame
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	6 months
Secondary Endpoint

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

3 Years

Maximum Age

14 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Label	URL
Edison Pharmaceuticals Website	View source

Condition Browse Module

MeSH Terms

ID	Term
D000067877	Autism Spectrum Disorder
D001321	Autistic Disorder
D028361	Mitochondrial Diseases
D015518	Rett Syndrome
D017204	Angelman Syndrome

Ancestor Terms

ID	Term
D002659	Child Development Disorders, Pervasive
D065886	Neurodevelopmental Disorders
D001523	Mental Disorders
D008659	Metabolic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided