Trial · NCT02594124
Registered outcomes32
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
21 to 32 of 32
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Secondary | Change From Baseline in Weight for Age Percentile | MMDR Period: up to Day 1800 | Participants who were below the age of 36 months were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. The weight for age percentile was calculated using either WHO or CDC scales. The CDC scale allows to calculate the weight for age percentile up to 20 years, while the WHO scale allows to calculate it only up to 10 years. |
| Secondary | Percentage of CMAP Responders | MMDR Period: At Day 1800 | CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. A participant was defined as a responder if they had a peroneal amplitude ≥1 mV at last visit (including the amplitude ≥1 mV at baseline and also demonstrated as such at last visit). |
| Secondary | Number of Participants Who Achieved Motor Milestones | MMDR Period: up to Day 1800 | Motor milestones were measured based on WHO criteria. The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support (SWS), standing with assistance (SWA), hands and knees crawling (HKC), and walking alone (WA). |
| Secondary | Number of Participants Who Achieved Standing Alone and Walking With Assistance | MMDR Period: up to Day 1800 | Motor milestones were measured based on WHO criteria. The WHO motor milestones are a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: SWS, SWA, HKC, WWA, WA and standing alone (SA). SA and WWA were assessed in this outcome measure. |
| Secondary | Total Number of Hospitalizations Due to Serious Respiratory Events | Up to day 2520 | Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization. |
| Secondary | Total Number of Hospitalizations Due to Serious Adverse Events | Up to day 2520 | Total number of hospitalizations is total number of serious events that occurred during study for all participants under each group. For a participant with multiple SAEs which started at the same date and led to hospitalization, it is counted as one hospitalization. |
| Secondary | Percent of Time in Hospitalization | Upto day 2160 | |
| Secondary | Change From Baseline in Cobb-Angle on X-Ray of the Thoracolumbar Spine by Visit | MMDR Period: Baseline, MMDR Day 1800 | Cobb angle is a measurement of the degree of side-to-side spinal curvature used to define scoliosis. |
| Secondary | Pediatric Quality of Life Inventory (PedsQL) Questionnaires Total Score by Domain | MMDR Period: At Day 1800 | Items on the PedsQL generic core scale are reverse scored and transformed to a 0-100 scale. The PedsQL parent (P) and self (S) reported questionnaire was collected for participants from 2 to 25 years of age. Four dimensions were collected: Physical, Emotional, Social and School functioning and each item was scored on a 5 point ordinal scale. 0 (never) =100, 1 (almost never) = 75, 2 (sometimes)= 50, 3 (often) = 25, 4 (almost always) = 0. A total score was calculated as the sum of all the items over the number of items answered on all the scales. If more than 50% of items or more were missing, the scale score was not computed. Higher scores indicated better health related quality of life. |
| Secondary | Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) Questionnaire Total Score | MMDR Period: up to Day 1800 | The ACEND is a questionnaire that includes a total of seven domains assessing physical impact (including feeding/grooming/dressing, sitting/play, transfers, and mobility) and general caregiver impact (including time, emotion, and finance) and each domain comprises several items. The total score (TS) for each domain will be calculated on a scale of 0 to 100. Higher scores indicate a greater impact on the caregiver. |
| Secondary | Number of Participants With Disease-related Hospitalizations and AEs | MMDR Period: up to Day 1800 | |
| Secondary | Survival Rate | MMDR Period: up to Day 1800 | Survival rate was defined as the percentage of participants alive during the study and was estimated from the Kaplan Meier (KM) curve for time to death. |
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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