Trial · NCT03779334
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 28
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excluding the Known SMN2 Gene Modifier Mutation c.859G>C) and Baseline Compound Muscle Action Potential (CMAP) >=1.5 Millivolt (mV) Who Are Sitting Without Support | At Month 12 | The Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. An exact binomial test was performed. If the lower limit of the two-sided 90% CI was above the 5% threshold, the primary objective of the study was considered achieved. |
| Secondary | Percentage of Participants Developing Clinically Manifested SMA | At Month 12 and 24 | |
| Secondary | Time to Permanent Ventilation and/or Death | Up to 7 years | |
| Secondary | Percentage of Participants Who Are Alive Without Permanent Ventilation | At Month 12 and 24 | |
| Secondary | Percentage of Participants Alive | At Month 12 and 24 | |
| Secondary | Percentage of Participants Who Achieve the Attainment Level of the Motor Milestones as Assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2) | At Month 12 and 24 | HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking |
| Secondary | Percentage of Participants With Two Copies of the SMN2 Gene Sitting Without Support for 5 Seconds (Independent of the CMAP Value at Baseline). | At Month 12 | Assessed in Item 22 of the BSID-III Gross Motor Scale. The BSID-III is a commonly used measure of infant and toddler development (0 to 42 months). The normed-scores derived from the BSID-III are used in clinical practice to detect infants with developmental delays, as well as to evaluate developmental progress and the impact of therapeutic interventions. The gross motor scale consists of 72 items scored at 0 (unable to perform the activity) or 1 (criteria for item achieved). Item 22, "sits without support for 5 seconds", is not considered achieved if the infant sits alone for less than 5 seconds before losing balance and falling over, or if the infant uses his or her arms to prop him- or herself up. 90% CI for one sample binomial was computed using Clopper-Pearson (exact) method. |
| Secondary | Percentage of Participants Sitting Without Support for 5 Seconds | At Month 24 | Assessed with BSID-III Gross Motor Scale |
| Secondary | Percentage of Participants Sitting Without Support for 30 Seconds | At Month 12 and 24 | Assessed with BSID-III Gross Motor Scale |
| Secondary | Percentage of Participants Standing for at Least 3 Seconds | At Month 24 | Assessed with BSID-III Gross Motor Scale |
| Secondary | Percentage of Participants Walking (Takes at Least 3 Steps) | At Month 24 | Assessed with BSID-III Gross Motor Scale |
| Secondary | Percentage of Participants Demonstrating the Ability to Achieve a Scaled Score on BSID-III Gross Motor Subtests Within 1.5 Standard Deviations of Chronological Reference Standard | At Month 24 and 42 | Assessed through BSID-III Gross Motor Scale |
| Secondary | Change From Baseline Score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale at Month 12 | Baseline, Month 12 | The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. A positive change from baseline indicates an improvement. |
| Secondary | Percentage of Participants Who Achieve a Score of 40 or Higher, 50 or Higher, and 60 or Higher in the CHOP INTEND Motor Function Scale at Month 12 | At Month 12 | The CHOP-INTEND is a measure of motor function that was developed from the Test of Infant Motor Performance specifically for weak infants with neuromuscular disease. It consists of 16 items, where each item assesses a specific motor task (such as spontaneous movement of upper and lower extremity, hand grasping, rolling, head control, and others) graded on a scale of 0 to 4, where zero is no response and 4 is a complete response. A total score is calculated by summing the item scores (range 0 to 64) with lower scores indicating greater severity. Data are presented with a two-sided 90% Clopper-Pearson (exact) CI for the proportion. |
| Secondary | Percentage of Participants Who Meet CHOP INTEND Stopping Criteria at Any Point | Up to Month 24 | |
| Secondary | Change From Baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) Score | At Month 60 | |
| Secondary | Number and Percentage of Participants Within 3rd Percentile of Normal Range for Weight-for-Age, Length/Height-for-Age and Weight-for-Length/Height | At Month 12, 24, 36, 48 and 60 | Based on the WHO Child Growth Standards (WHO 2019) |
| Secondary | Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age | At Month 12 and 24 | Based on the WHO Child Growth Standards (WHO 2019) |
| Secondary | Change From Baseline Percentiles for Weight-for-age, Length/Height-for-age, and weight-for- Length/Height | At Month 12, 24, 36, 48 and 60 | |
| Secondary | Change From Baseline Percentiles for Head Circumference- For-age | At Month 12 and 24 |
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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