Trial · NCT03032172
Outcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 12 of 12
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events (AEs) | Baseline up to 5 years | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Discontinued Treatment Due to AEs | Baseline up to 5 years | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Shift in Puberty Status From Baseline | Baseline, Week 52, 104, 156, 208 and 260 | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. No participants from the AVXS-101 arm were assessed for Tanner staging since all participants in this arm were under 9 years of age. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Protocol-defined Neurological Conditions (NC) | Baseline up to Week 260 | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Emergence or Worsening of Symptoms as Assessed Using Columbia Suicide Severity Rating Scale (C-SSRS) | Up to approximately 5 years | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Participants with SI, suicidal behavior, and self-injurious behavior without suicidal intent are reported for this outcome measure. Overall number analyzed are the number of participants with at least one post-baseline assessment. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Anthropometric Examination: Change From Baseline in Weight | Baseline up to Week 260 | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint. | kilograms (kg) | MEAN |
| PRIMARY | Anthropometric Examination: Change From Baseline in Height | Baseline up to Week 260 | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint. | centimeters (cm) | MEAN |
| PRIMARY | Anthropometric Examination: Change From Baseline in Head Circumference | Baseline up to Week 208 | SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint. | cm | MEAN |
| PRIMARY | Maximum Plasma Concentration (Cmax) of Risdiplam | Predose on Weeks 2, 4, 13, 26, 39, 52, 65, 91 and post-dose on Weeks 1, 4, 13, 52, 91 and 104 | PK population included all participants with at least one timepoint with a measurable drug concentration. Overall number analyzed is the number of participants with data available for analysis. | nanograms per milliliter (ng/mL) | MEDIAN |
| PRIMARY | Area Under the Concentration-Time Curve (AUC0-24h) of Risdiplam | 24 hours Postdose at Year 2 Visit | PK population included all participants with at least one timepoint with a measurable drug concentration. Overall number analyzed is the number of participants with data available for analysis. | nanograms*hour/milliliter (ng*h/mL) | MEDIAN |
| PRIMARY | Plasma Trough Concentration (Ctrough) of Risdiplam | Predose at Year 5 visit | PK population included all participants with at least one timepoint with a measurable drug concentration. Overall number analyzed is the number of participants with data available for analysis. | ng/mL | MEDIAN |
| SECONDARY | Survival of Motor Neuron (SMN) Protein Levels in Blood | Baseline and Year 5 | Pharmacodynamic (PD) population included all participants with at least one timepoint with a measurable PD marker. Overall number analyzed is the number of participants with data available for analysis. | ng/mL | MEDIAN |
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
Read this list from software.
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GET https://conductscience.org/api/trials/v1/NCT03032172/outcomes