Trial · NCT03039686
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 17 of 17
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Baseline for the North Star Ambulatory Assessment (NSAA) Total Score | Baseline | The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. |
| Primary | Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48 | Baseline, Week 48 | The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM). |
| Secondary | Baseline Time for 4 Stair Climb | Baseline | The time to complete the 4 stair climb was measured at baseline. |
| Secondary | Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV) | Baseline, Week 48 | 4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM). |
| Secondary | Baseline for the Time to Stand From Supine | Baseline | The time required for a participant to stand from supine position. A longer time reflects a worse outcome. |
| Secondary | Change From Baseline at Week 48 in Stand From Supine Velocity | Baseline, Week 48 | The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM). |
| Secondary | Baseline Time for 10 Meter Walk/Run | Baseline | The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome. |
| Secondary | Change From Baseline at Week 48 in 10 M Walk/Run Velocity | Baseline, Week 48 | The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM). |
| Secondary | Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale | Baseline | The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. |
| Secondary | Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale | Baseline, Week 48 | The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM). |
| Secondary | Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength | Baseline, Week 48 | Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement. |
| Secondary | Baseline for the 6 Minute Walk Distance (6MWD) | Baseline | The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. |
| Secondary | Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD) | Baseline, Week 48 | The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM). |
| Secondary | Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48 | Baseline, Week 48 | The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse" at Week 48 as compared to baseline. |
| Secondary | Change From Baseline at Week 48 in 95th Percentile Stride Velocity | Baseline, Week 48 | Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement. |
| Secondary | Number of Participants With Adverse Events (AEs) | During DB period (48 weeks) and Whole study (up to approximately 34 months) | An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. |
| Secondary | Number of Participants With AEs Leading to Discontinuation | During DB period (48 weeks) and Whole study (up to approximately 34 months) | An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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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