Phase 2 Study of EPI-743 in Children With Pearson Syndrome | NCT02104336

Studies
Phase 2 Study of EPI-743 in Children With Pearson Syndrome

NCT02104336

Sponsor

Edison Pharmaceuticals Inc

Status

Terminated

Last Update Posted

Nov 18, 2020Actual

Enrollment

2Actual

Phase

Phase 2

Conditions

Pearson Syndrome

Interventions

EPI-743

Countries

United States

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

Nov 2020

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Results from other studies did not support continuation of this trial

Expanded Access Info

Start Date

Aug 31, 2014Actual

Primary Completion Date

Nov 30, 2015Actual

Completion Date

Feb 29, 2016Actual

First Submitted Date

Apr 2, 2014

First Submission Date that Met QC Criteria

Apr 3, 2014

First Posted Date

Apr 4, 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

Aug 17, 2020

Certification/Extension First Submitted that Passed QC Review

Aug 24, 2020

Certification/Extension First Posted Date

Aug 31, 2020Actual

Last Update Submitted Date

Nov 16, 2020

Last Update Posted Date

Nov 18, 2020Actual

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

Description Module

Brief Summary

Treatment of Pediatric Subjects with Pearson syndrome

Detailed Description

If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.

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

2Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
EPI-743	Experimental	15 mg/kg EPI-743 to be administered three times per day for 1 year	Drug: EPI-743

Interventions

Name	Type	Description	Arm Group Labels	Other Names
EPI-743	Drug	EPI-743 is the quinone oxidation product of alpha-tocotrienol	EPI-743

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure		1 year

Secondary Outcomes

Measure	Description	Time Frame
Transfusion avoidance		1 year
Hematologic function		1 year
Neuromuscular function

Other Outcomes

Measure	Description	Time Frame
Number of Dose Limiting SAEs	Any adverse event greater than or equal to grade 3 that is deemed related to treatment with EPI-743 and not related to underlying disease	1 year

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Genetically confirmed diagnosis of Pearson syndrome
Age less than 18
Availability of medical history for 12 months prior to enrollment
Abstention from use of CoQ10, vitamin E, lipoic acid and Idebenone 14 days prior to treatment with EPI-743

Exclusion Criteria:

Allergy to EPI-743, sesame oil or vitamin E
Clinical history of bleeding/ abnormal PT/PTT
Concurrent inborn errors of metabolism
Use of anticoagulant medications
Participation in any interventional study within 30 days of treatment
Use of erythropoietin 30 days prior to trial enrollment

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Matthew Klein, MD, FACS	Edison Pharmaceuticals	Study Chair

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Orange County		Orange	California	82868	United States
Cleveland Clinic

Condition Browse Module

MeSH Terms

ID	Term
C536353	VLCAD deficiency
D028361	Mitochondrial Diseases

Ancestor Terms

ID	Term
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C571746	alpha-tocotrienol quinone

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided