Trial · NCT03779334
Outcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 20 of 28
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| 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 | Primary efficacy population included all infants in the intent-to-treat (ITT) population with two SMN2 copies (excluding the known SMN2 gene modifier mutation c.859G\>C) and a baseline compound muscle action potential (CMAP) amplitude \>/= 1.5 mV. | percentage of participants | NUMBER |
| 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 | |||
| 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 | Intent-to-treat (ITT) population included all enrolled participants, regardless of whether they received risdiplam or not. Only participants with two copies of the SMN2 gene were included in this outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Sitting Without Support for 5 Seconds | At Month 24 | |||
| SECONDARY | Percentage of Participants Sitting Without Support for 30 Seconds | At Month 12 and 24 | |||
| SECONDARY | Percentage of Participants Standing for at Least 3 Seconds | At Month 24 | |||
| SECONDARY | Percentage of Participants Walking (Takes at Least 3 Steps) | At Month 24 | |||
| 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 | |||
| 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 | ITT population included all enrolled participants, regardless of whether they received risdiplam or not. | score on a scale | MEDIAN |
| 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 | ITT population included all enrolled participants, regardless of whether they received risdiplam or not. | percentage of participants | NUMBER |
| 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 | |||
| SECONDARY | Number and Percentage of Participants Within 3rd Percentile of Normal Range for Head Circumference-for-age | At Month 12 and 24 | |||
| 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.
For software
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GET https://conductscience.org/api/trials/v1/NCT03779334/outcomes