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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 is a Phase 1 study that has completed, run by National Institute of Nursing Research (NINR). The registered enrollment target is 63 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02362425
The ClinicalTrials.gov registry entry for NCT02362425 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 63 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Institute of Nursing Research (NINR), which has 46 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT02362425 reports 0 study locations.
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.
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