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NCT02362425 · ClinicalTrials.gov registry record · Phase 1
Antioxidant Therapy in RYR1-Related Congenital Myopathy
A Phase 1 study, sponsored by National Institute of Nursing Research (NINR).
- Completed
- Registry status
- Phase 1
- Development phase
- 63
- Enrollment target
NCT02362425: Completed Phase 1 study, sponsored by National Institute of Nursing Research (NINR).
NCT02362425 is a Phase 1 study that has completed, run by National Institute of Nursing Research (NINR). The registered enrollment target is 63 participants, roughly in line with the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02362425, a Phase 1 study, has completed, sponsored by National Institute of Nursing Research (NINR).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 63 participants
- Enrollment target
Study Summary
Background: \- Ryanodine receptor type 1-related myopathies (RYR1-RM) are the most common non-dystrophic muscle diseases that people are born with in the U.S. They affect development, muscles, and walking. Researchers want to test a new drug to help people with these diseases. Objectives: \- To see if the drug N-acetylcysteine decreases muscle damage in people with RYR1-RM. To see if it improves their exercise tolerance. Eligibility: \- People age 7 and older with a confirmed genetic diagnosis of RYR1 or a clinical diagnosis of RYR1 and a family member with a confirmed genetic diagnosis. Design: * Participants will be screened with a checklist of criteria. Adult participants may have a muscle biopsy. A needle will remove a tiny piece of muscle in the lower leg. * Study visits will take several days. * Visit 1: * Medical history * Physical exam * Blood, urine, and saliva tests * Questions about symptoms and quality of life * Heart, lung, and walking tests * Muscle Oxygenation Capacity Test. A blood pressure cuff around the thigh will be tightened for up to 10 minutes. * Biodex testing, stretching the leg against resistance * Muscle ultrasounds. A probe will be moved over the skin. * Participants may be photographed or videotaped during procedures. * They may have a muscle biopsy. * Six months later, visit 2 will repeat visit 1. Participants will start taking the study drug dissolved in water or placebo three times a day for 6 months. * Participants will stay at NIH for 2 days after starting the study drug. * Participants will be contacted by phone during the study to monitor side effects * Six months after starting the study drug, study visit 3 will repeat some or all of visit 1.
Primary Outcome
Urine will be assayed for 15-isoprostane-F2, which is formed when arachidonic acid reacts with reactive oxygen species(ROS). A validated gas chromatography(GC)-mass spectrometer (MS) method will be used to quantify 15-isoprostane-F2
Interventions
- DRUG Placebo
- DRUG N-acetylcysteine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 63 participants |
| Start Date | 2015-02-12 |
| Est. Completion | 2018-05-30 |
| Phase | Phase 1 |
What the finished NCT02362425 record still lists
NCT02362425 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 63 participants, a relatively small participant target, roughly in line with the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT02362425 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02362425 about?
NCT02362425 is a clinical study titled "Antioxidant Therapy in RYR1-Related Congenital Myopathy". Background: \- Ryanodine receptor type 1-related myopathies (RYR1-RM) are the most common non-dystrophic muscle diseases that people are born with in the U.S. They affect development, muscles, and walking. Researchers want to test a new drug to help people with these diseases. Objectives: \- To s...
What is the current status of trial NCT02362425?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 63 participants. The study started on 2015-02-12. Estimated completion is 2018-05-30.
What interventions are being tested in trial NCT02362425?
The interventions under investigation include: Placebo (DRUG), N-acetylcysteine (DRUG).
Who is sponsoring clinical trial NCT02362425?
This trial is sponsored by National Institute of Nursing Research (NINR), which has 46 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02362425's enrollment target sits among peer trials
63 820th of 2000 higher than 1,177 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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