Trials / No Longer Available
No Longer AvailableNCT01370447
EPI-743 for Mitochondrial Respiratory Chain Diseases
Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life Care
- Status
- No Longer Available
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- PTC Therapeutics · Industry
- Sex
- All
- Age
- 1 Year
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | EPI-743 | EPI-743 (oral solution \[100 mg/mL\] or liquid-filled capsules \[100 mg or 200 mg\]) will be administered per dose and schedule specified in the arm. |
Timeline
- First posted
- 2011-06-10
- Last updated
- 2024-01-23
Locations
16 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT01370447. Inclusion in this directory is not an endorsement.