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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 is a Phase 1 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 20 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01642056
The ClinicalTrials.gov registry entry for NCT01642056 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Human Genome Research Institute (NHGRI), which has 185 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01642056 reports 0 study locations.
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.
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