Trial · NCT02362425
Antioxidant Therapy in RYR1-Related Congenital Myopathy
NCT02362425, Phase 1, Phase 2, Completed, National Institute of Nursing Research (NINR); results first posted 2019-12-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Antioxidant Therapy in RYR1-Related Congenital Myopathy. Trials, record NCT02362425. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02362425
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Antioxidant Therapy in RYR1-Related Congenital Myopathy
- NCT number
- NCT02362425
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 63 participants, actual
- Lead sponsor
- National Institute of Nursing Research (NINR)
- Conditions
- Neuromuscular Disease
- Interventions
- N-acetylcysteine, Placebo
- First posted
- 2015-02-13
- Last updated
- 2019-12-24
- Results first posted
- 2019-12-24
Source ClinicalTrials.gov
Registered outcomes56
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Urine 15-F2t Isoprostane Concentration | 12 months | 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 |
| Primary | Six Minute Walk Test (6MWT) | 12 months | Meters walked in 6 minutes will be recorded; distances in meters will be recorded at each minute interval; speed will be calculated. |
| Secondary | DCF-fluorescence Intensity (AU) | 12 months | Dichlorodihydrofluorescein (DCFH) will be used on participate muscle biopsies to analyze intracellular oxidant activity \[H2DCFDA (H2-DCF, DCF)\]. |
| Secondary | Time to Ascend Steps (Seconds) | 12 months | Participants completed the following timed function tests: supine to stand, ascend four steps, descend four steps, and walk/run 10 meters. For time taken to ascend four steps, the subject was asked to ascend four steps whilst being timed. |
| Secondary | Descend Steps | 12 months | Participants completed the following timed function tests: supine to stand, ascend four steps, descend four steps, and walk/run 10 meters. For time taken to descend four steps, the subject was asked to descend four steps whilst being timed. |
| Secondary | Walk/Run 10 Meters | 12 months | Participants completed the following timed function tests: supine to stand, ascend four steps, descend four steps, and walk/run 10 meters. For walk/run 10 meters, participants were timed as they walked/ran a marked 10-meter course as quickly as possible. |
| Secondary | Supine to Stand | 12 months | Participants completed the following timed function tests: supine to stand, ascend four steps, descend four steps, and walk/run 10 meters. For time taken to transition from supine to standing, participants were asked to lay supine and then the time taken to move from supine to standing was recorded. |
| Secondary | Motor Function Measure-32 (MFM-32) Domain 1 (D1) | 12 months | Motor Function Measure, MFM-32 is a well-established scale of motor function in congenital muscle disease. The D1 domains measure standard and transfers, and consist of 13 items. Generic Values for each domain are: 0 = cannot perform the task, 1 = initiated the task, 2 - performs the movement incompletely, or completely but imperfectly, 3 = performs the task fully and 'normally'. Total score is the sum of all the domains for D1 divided by the maximum score possible and multiplied by 100. Min=0, Max = 100. Lower numbers on the scale represent a worse outcome. |
| Secondary | Motor Function Measure-32 (MFM-32) Domain 2 (D2) | 12 months | Motor Function Measure, MFM-32 is a well-established scale of motor function in congenital muscle disease. The D2 domains measure axial and proximal motor function and consists of 12 items. Generic Values for each domain are: 0 = cannot perform the task, 1 = initiated the task, 2 - performs the movement incompletely, or completely but imperfectly, 3 = performs the task fully and 'normally'. The total score is the sum of all the MFM-32 domains for D2, divided by maximum score possible and multiplied by 100. Min = 0, Max = 100. Lower numbers on the scale represent a worse outcome. |
| Secondary | Motor Function Measure-32 (MFM-32) Domain 3 (D3) | 12 months | Motor Function Measure, MFM-32 is a well-established scale of motor function in congenital muscle disease. The D3 domains measure distal motor function and consist of 7 items. Generic Values for each domain are: 0 = cannot perform the task, 1 = initiated the task, 2 - performs the movement incompletely, or completely but imperfectly, 3 = performs the task fully and 'normally'. The total score is the sum of all the MFM-32 domains for D3, divided by maximum score possible and multiplied by 100. Min = 0, Max = 100. Lower numbers on the scale represent a worse outcome. |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 registered outcomesOutcomes56
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Urine 15-F2t Isoprostane Concentration | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers post-baseline (months 6 and 12), thus they are not included in the analysis. | ng/mg Cr | LEAST_SQUARES_MEAN |
| PRIMARY | Six Minute Walk Test (6MWT) | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | meters | LEAST_SQUARES_MEAN |
| SECONDARY | DCF-fluorescence Intensity (AU) | 12 months | This analysis was performed only on participants in the randomized population who volunteered to have the muscle biopsy performed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | AU | MEAN |
| SECONDARY | Time to Ascend Steps (Seconds) | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Descend Steps | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Walk/Run 10 Meters | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Supine to Stand | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Motor Function Measure-32 (MFM-32) Domain 1 (D1) | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | % of maximum score | LEAST_SQUARES_MEAN |
| SECONDARY | Motor Function Measure-32 (MFM-32) Domain 2 (D2) | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | % of maximum score | LEAST_SQUARES_MEAN |
| SECONDARY | Motor Function Measure-32 (MFM-32) Domain 3 (D3) | 12 months | Outcome measures are reported for participants who were randomized and continued into the randomized, double-blind period of the trial. Missing data was not imputed. This measure was not collected for Healthy Volunteers, thus they are not included in this analysis. | % of maximum score | LEAST_SQUARES_MEAN |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 outcomesResults by group112
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Urine 15-F2t Isoprostane Concentration | N-acetylcysteine (NAC) | 2.7 | 95% Confidence Interval 1.7 to 3.7 | ng/mg Cr | |
| Urine 15-F2t Isoprostane Concentration | Placebo | 2.6 | 95% Confidence Interval 1.5 to 3.6 | ng/mg Cr | |
| Six Minute Walk Test (6MWT) | N-acetylcysteine (NAC) | 495.8 | 95% Confidence Interval 475.8 to 515.9 | meters | |
| Six Minute Walk Test (6MWT) | Placebo | 471.9 | 95% Confidence Interval 451.1 to 492.7 | meters | |
| DCF-fluorescence Intensity (AU) | N-acetylcysteine (NAC) | 1.9 | 95% Confidence Interval 0.7 to 3.1 | AU | |
| DCF-fluorescence Intensity (AU) | Placebo | 3.4 | 95% Confidence Interval 1.7 to 5.1 | AU | |
| Time to Ascend Steps (Seconds) | N-acetylcysteine (NAC) | 3.2 | 95% Confidence Interval 2.9 to 3.5 | seconds | |
| Time to Ascend Steps (Seconds) | Placebo | 3.3 | 95% Confidence Interval 3.0 to 3.6 | seconds | |
| Descend Steps | N-acetylcysteine (NAC) | 1.9 | 95% Confidence Interval 1.5 to 2.3 | seconds | |
| Descend Steps | Placebo | 2.4 | 95% Confidence Interval 2.1 to 2.8 | seconds |
1 to 10 of 112Source ClinicalTrials.gov
See all 112 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Phase 1 (up to 6 Months) | STARTED | RYR1-RM Volunteers | 53 | |
| Phase 1 (up to 6 Months) | STARTED | Healthy Volunteers | 10 | |
| Phase 1 (up to 6 Months) | STARTED | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 0 | |
| Phase 1 (up to 6 Months) | STARTED | RYR1-RM Volunteers: Placebo | 0 | |
| Phase 1 (up to 6 Months) | COMPLETED | RYR1-RM Volunteers | 37 | |
| Phase 1 (up to 6 Months) | COMPLETED | Healthy Volunteers | 10 | |
| Phase 1 (up to 6 Months) | COMPLETED | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 0 | |
| Phase 1 (up to 6 Months) | COMPLETED | RYR1-RM Volunteers: Placebo | 0 | |
| Phase 1 (up to 6 Months) | NOT COMPLETED | RYR1-RM Volunteers | 16 | |
| Phase 1 (up to 6 Months) | NOT COMPLETED | Healthy Volunteers | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events276
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Blurred Vision | Eye disorders | RYR1-RM Volunteers | 0 | 53 | |
| other | Blurred Vision | Eye disorders | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 1 | 16 | |
| other | Blurred Vision | Eye disorders | RYR1-RM Volunteers :Placebo | 0 | 17 | |
| other | Blurred Vision | Eye disorders | Healthy Volunteers | 0 | 10 | |
| other | Barrett's Oesophagus | Gastrointestinal disorders | RYR1-RM Volunteers | 0 | 53 | |
| other | Barrett's Oesophagus | Gastrointestinal disorders | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 1 | 16 | |
| other | Barrett's Oesophagus | Gastrointestinal disorders | RYR1-RM Volunteers :Placebo | 0 | 17 | |
| other | Barrett's Oesophagus | Gastrointestinal disorders | Healthy Volunteers | 0 | 10 | |
| other | Stomach Virus | Gastrointestinal disorders | RYR1-RM Volunteers | 0 | 53 | |
| other | Stomach Virus | Gastrointestinal disorders | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 0 | 16 |
1 to 10 of 276Source ClinicalTrials.gov
See all 276 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RYR1-RM Volunteers | 0 | 53 |
| Total, all-cause mortality | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 0 | 16 |
| Total, all-cause mortality | RYR1-RM Volunteers :Placebo | 0 | 17 |
| Total, all-cause mortality | Healthy Volunteers | 0 | 10 |
| Total, other adverse events | RYR1-RM Volunteers | 36 | 53 |
| Total, other adverse events | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 16 | 16 |
| Total, other adverse events | RYR1-RM Volunteers :Placebo | 17 | 17 |
| Total, other adverse events | Healthy Volunteers | 5 | 10 |
| Total, serious adverse events | RYR1-RM Volunteers | 1 | 53 |
| Total, serious adverse events | RYR1-RM Volunteers: N-acetylcysteine (NAC) | 4 | 16 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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