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Trial · NCT03779334

Registered outcomes28

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
SecondaryChange From Baseline in Chest CircumferenceAt Month 12 and 24
SecondaryRatio Between Chest and Head CircumferencesAt Month 12 and 24
SecondaryPercentage of Participants With the Ability to Swallow and to Feed OrallyAt Month 12, 24, 36, 48 and 60
SecondaryChange From Baseline in Compound Muscle Action Potential (CMAP) AmplitudeAt Month 12 and 24Measured by CMAP
SecondaryMeasurement of Pharmacodynamic Marker Levels in BloodDay 1, 56, 196, 364, 728 and at early withdrawal
SecondaryPercentage of Participants With Adverse EventsUp to 7 yearsAdverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5
SecondaryOphthalmological Examination as Appropriate for AgeUp to 7 years
SecondaryPlasma Concentration of Risdiplam and Its Metabolites to Characterize the PK ProfileUp to 7 years

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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