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Trial · NCT05115110

Registered outcomes34

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

ListOutcomeTime frameDescription
PrimaryPart 1 - Percentage of participants with adverse events (AEs)Up to 4.5 years
PrimaryPart 1 - Incidence of relevant echocardiographic parameter z scores > 2Up to 4.5 years
PrimaryPart 1 - Serum concentration of RO7204239Through Week 96
PrimaryPart 1 - Time to maximum serum concentration (Cmax) of RO7204239Through Week 96
PrimaryPart 1 - Area under the curve (AUC) of RO7204239Through Week 96
PrimaryPart 1 - Trough concentration (Ctrough) of RO7204239Through Week 96
PrimaryPart 1 - Plasma concentration of risdiplamWeek 21
PrimaryPart 1 - Plasma concentration of risdiplam metabolite (M1)Week 21
PrimaryPart 1 - Cmax of risdiplamWeek 21
PrimaryPart 1 - AUC of risdiplamWeek 21
PrimaryPart 1 - Ctrough of risdiplamWeek 21
PrimaryPart 1 - Incidence of anti-drug antibodies (ADAs)Through Week 96
PrimaryPart 1 - Change from baseline in serum concentration of total myostatinThrough Week 85
PrimaryPart 1 - Change from baseline in serum concentration of free latent myostatinThrough Week 85
PrimaryPart 1 - Change from baseline in serum concentration of mature myostatinThrough Week 85
PrimaryPart 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant thigh muscles as assessed by magnetic resonance imaging (MRI) in participants aged at least 5 yearsWeek 24 of combination treatment
PrimaryPart 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant calf muscles as assessed by MRI in participants aged at least 5 yearsWeek 24 of combination treatment
PrimaryPart 2 - Change from baseline in Revised Hammersmith Scale (RHS) total scoreWeek 72 of combination treatment (study Week 80)
SecondaryPart 2 - Change from baseline in Motor Function Measure (MFM) Domain 1 + Domain 2 (D1 + D2) scoreWeek 72 of combination treatment (study Week 80)
SecondaryPart 2 - Change from baseline in MFM-32 total scoreWeek 72 of combination treatment (study Week 80)

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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GET https://conductscience.org/api/trials/v1/NCT05115110/registered