Trial · NCT02908685
Outcomes33
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 20 of 33
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 1: Selected Part 2 Dose of Risdiplam for Participants With a Body Weight (BW) of >/=20kg | Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017) | All participants in Part 1. | milligram (mg) | NUMBER |
| PRIMARY | Part 1: Selected Part 2 Dose of Risdiplam for Participants With BW of <20kg | Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017) | All participants in Part 1. | milligram/kilogram (mg/kg) | NUMBER |
| PRIMARY | Part 2: Change From Baseline in the Total Motor Function Measure 32 (MFM-32) Total Score at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Percentage of Participants With Marked Improvement (Defined as >= 3) in the Total Motor Function Measure (MFM32) Score at Month 12 | At Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis. Missing results at Month 12 are considered as non-responders. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Change From Baseline in the Total Score of the Revised Upper Limb Module (RULM) at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing RULM total score at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in Total Score of Hammersmith Functional Motor Scale Expanded (HFMSE) at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in Forced Vital Capacity (FVC) at Month 12 in Participants Aged 6-25 Years | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing FVC data at Baseline were not included in the analysis. | Percentage Predicted | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the Caregiver-Reported SMA Independence Scale (SMAIS) Total Score at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing SMAIS total score at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Percentage of Participants Rated by Clinicians as Improved in the Clinical Global Impression of Change (CGI-C) Scale Ratings at Month 12 | At Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Missing results at Month 12 are considered as non-responders. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Participants Who Achieve Stabilization or Improvement (Defined as >= 0) in the Total Motor Function Measure (MFM-32) Score at Month 12 | At Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis. Missing results at Month 12 are considered as non-responders. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Percentage of Participants Who Achieve an Improvement of at Least One Standard Error of Measurement on the Total MFM-32 Score at Month 12 | At Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis. | Percentage of Participants | NUMBER |
| SECONDARY | Part 2: Change From Baseline in the MFM-32 Domain 1 (D1) Score at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D1 score at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the MFM-32 Domain 2 (D2) Score at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D2 score at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the MFM-32 Domain 3 (D3) Score at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D3 score at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the Total Combined Scores of MFM-32 Domains 1 and 2 at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D1+D2 scores at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the Total Combined Scores of MFM-32 Domains 2 and 3 at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D2+D3 scores at Baseline were not included in the analysis. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 12 in Participants Aged 6-25 Years | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing FEV1 data at Baseline were not included in the analysis. | Percentage Predicted | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the Peak Cough Flow (PCF) at Month 12 in Participants Aged 6-25 Years | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing PCF data at Baseline were not included in the analysis. | Percent Predicted | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in the Best Sniff Nasal Inspiratory Pressure (SNIP) at Month 12 | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing SNIP data at Baseline were not included in the analysis. | Percentage Predicted | LEAST_SQUARES_MEAN |
| SECONDARY | Part 2: Change From Baseline in Maximal Inspiratory Pressure (MIP) at Month 12 in Participants Aged 6-25 Years | Baseline (Day-1) and Month 12 | The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MIP data at Baseline were not included in the analysis. | Percentage Predicted | LEAST_SQUARES_MEAN |
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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