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NCT01642056 · ClinicalTrials.gov registry record · Phase 1
EPI-743 for Metabolism or Mitochondrial Disorders
A Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
NCT01642056: Completed Phase 1 study, sponsored by National Human Genome Research Institute (NHGRI).
NCT01642056 is a Phase 1 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 20 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01642056, a Phase 1 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
Study Summary
Background: * Mitochondria are the parts of cells that help produce energy. Metabolism is the process by which the body uses energy to help cells grow and reproduce. Metabolic and mitochondrial disorders affect the body s ability to produce and store energy. These disorders can cause a wide variety of problems, but most often they affect the muscles and the brain, where energy requirements are high. Treatment is difficult because the exact source of the problem is hard to detect. * EPI-743 is a new drug that is based on vitamin E. Tests have shown that it can help improve the function of cells with mitochondrial problems. It may be able to treat people with genetic disorders that affect metabolism and mitochondria. Objectives: \- To see if EPI-743 can improve energy production and use in people with mitochondrial or metabolic disorders. Eligibility: \- Children between 2 and 11 years of age who have metabolic or mitochondrial problems. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. * The study will last about 13 months. Participants will have seven 3- to 5-day inpatient study visits about 3 months apart. * Participants will take either EPI-743 or a placebo for the first 6 months of the study. After 6 months, there will be a 1-month rest period. Then, those who received EPI-743 in the first 6 months will take the placebo for the next 6 months. Those who had the placebo will take EPI-743. * During each inpatient study visit, participants will have a physical exam. A 24-hour urine collection will be obtained. Blood samples will also be taken. Imaging studies and other tests may be performed as directed by the study researchers.
Primary Outcome
NPMDS is a semi-quantitative clinical rating scale that evaluates the progression of mitochondrial disease in pediatric patients. The NPMDS scale provides an assessment of the progression of mitochondrial disease. Scores from Parts I-III are added to get a value between 0 and 130, with higher scores representing more severe disease.
Interventions
- DRUG Placebo
- DRUG EPI-743
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2012-09-01 |
| Est. Completion | 2019-09-24 |
| Phase | Phase 1 |
What the finished NCT01642056 record still lists
NCT01642056 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01642056 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01642056 about?
NCT01642056 is a clinical study titled "EPI-743 for Metabolism or Mitochondrial Disorders". Background: * Mitochondria are the parts of cells that help produce energy. Metabolism is the process by which the body uses energy to help cells grow and reproduce. Metabolic and mitochondrial disorders affect the body s ability to produce and store energy. These disorders can cause a wide variety...
What is the current status of trial NCT01642056?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2012-09-01. Estimated completion is 2019-09-24.
What interventions are being tested in trial NCT01642056?
The interventions under investigation include: Placebo (DRUG), EPI-743 (DRUG).
Who is sponsoring clinical trial NCT01642056?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01642056's enrollment target sits among peer trials
20 1629th of 2000 higher than 285 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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