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EPI-743 for Mitochondrial Respiratory Chain Diseases

ClinicalTrials.gov ID: NCT01370447

Public ClinicalTrials.gov record NCT01370447. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 21, 2026, 7:39 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life Care

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.

Study identification

NCT ID
NCT01370447
Recruitment status
No Longer Available
Study type
Expanded access
Phase
Not listed
Lead sponsor
PTC Therapeutics
Industry
Enrollment
Not listed

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
1 Year and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Not listed
Primary completion
Not listed
Completion
Not listed
Last update posted
Jan 23, 2024

United States locations

U.S. sites
16
U.S. states
9
U.S. cities
15
Facility City State ZIP Site status
UCLA Los Angeles California 90095 —
CHOC Children's Clinic Orange California 92868 —
Lucille Packard Children's Hospital Palo Alto California 94304 —
UCSD San Diego California 92123 —
Stanford Children's Health Stanford California 94304 —
Emory University Decatur Georgia 30033 —
CUMC (Columbia University) New York New York 10032 —
Carolinas Medical Center Charlotte North Carolina 28203 —
Akron Children's Hospital Akron Ohio 44308 —
Cleveland Clinic Cleveland Ohio 44195 —
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104 —
MUSC North Charleston South Carolina 29406 —
Baylor College of Medicine Houston Texas 77030 —
UTH Houston Texas 77030 —
Naval Hospital, Bremerton Bremerton Washington 98312 —
Seattle Children's Hospital Seattle Washington 98105 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01370447, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jan 23, 2024 · Synced Oct 21, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT01370447 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →