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Trial · NCT01609062

Outcomes13

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

TypeOutcomeTime framePopulationUnitsMeasured as
PRIMARYSafety EvaluationEntire Study Period, up to 192 weeks or ETV (early termination visit)The primary outcome measure data is presented in more detail under the Adverse Events section.participantsNUMBER
SECONDARY6-minute Walk Test (6MWT)Baseline, Week 12, 24, and 52Modified Intent-to-Treat Analysis SetmetersMEAN
SECONDARY3-minute Stair Climb Test (3MSCT)Baseline, Week 12, 24, and 52Modified Intent-to-Treat Analysis Setstairs/minMEAN
SECONDARYRespiratory Function Test (MVV and FVC)Baseline, Week 12, 24, and 52Modified Intent-to-treat Analysis SetPercent ChangeMEAN
SECONDARYNormalized Urine Keratan Sulfate (uKS)Baseline, Week 12, 24, and 52Modified Intent-to-treat Analysis SetPercent ChangeMEAN
SECONDARYCardiopulmonary Exercise Testing (CPET) - Duration of ExerciseBaseline, Week 25 and 52A subset of mITT subjects who were scheduled to perform CPETPercent ChangeMEAN
SECONDARYCardiopulmonary Exercise Testing (CPET) - Peak WorkloadBaseline, Week 25 and 52A subset of mITT subjects who were scheduled to perform CPETPercent ChangeMEAN
SECONDARYCardiopulmonary Exercise Testing (CPET) - O2 PulseBaseline, Week 25 and 52A subset of mITT subjects who were scheduled to perform CPETPercent ChangeMEAN
SECONDARYCardiopulmonary Exercise Testing (CPET) - Aerobic EfficiencyBaseline, Week 25 and 52A subset of mITT subjects who were scheduled to perform CPETPercent ChangeMEAN
SECONDARYMuscle Strength Testing (MST) - Knee Extension TestBaseline, Week 25 and 52Modified Intent-to-treat Analysis SetPercent ChangeMEAN
SECONDARYMuscle Strength Testing (MST) - Knee Flexion TestBaseline, Week 25 and 52Modified Intent-to-treat Analysis SetPercent ChangeMEAN
SECONDARYMuscle Strength Testing (MST) - Elbow Flexion TestBaseline, Week 25 and 52Modified Intent-to-treat Analysis SetPercent ChangeMEAN
SECONDARYAdolescent Pediatric Pain Tool (APPT) - Pain IntensityBaseline, Week 12, 24, and 52Modified Intent-to-treatunits on a scaleMEAN

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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