EPI-743 for Mitochondrial Respiratory Chain Diseases
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.
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
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
Conditions
Interventions
- EPI-743 Drug
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 22, 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 22, 2024 · Synced Jul 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01370447 live on ClinicalTrials.gov.