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Trial · NCT03568175

Registered outcomes15

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

ListOutcomeTime frameDescription
PrimaryChange From Baseline in Heparan Sulfate Levels in Cerebrospinal FluidBaseline to 52 weeks
SecondaryChange From Baseline in Serum Heparan Sulfate Levels.Baseline, 24-26 weeks, 50-52 weeks
SecondaryChange From Baseline in Serum Dermatan Sulfate Levels.Baseline, 24-26 weeks, 50-52 weeks
SecondaryChange From Baseline in Urinary Heparan Sulfate Levels.Baseline, 25 weeks, 52 weeks
SecondaryChange From Baseline in Urinary Dermatan Sulfate Levels.Baseline, 25 weeks, 52 weeks
SecondaryChange From Baseline in Liver Volumes.Baseline, 25 weeks, 52 weeks
SecondaryChange From Baseline in Spleen Volumes.Baseline, 25 weeks, 52 weeks
SecondaryChange From Baseline in Cardiac Function.Baseline, 25 weeks, 52 weeks
SecondaryChange From Baseline in 6-minute Walk Test Distance.Baseline, 25 weeks, 52 weeksItem 9 will be administrated only in patients judged by the investigator or subinvestigator to be possible to perform the 6-minutes walk test
SecondaryChange From Baseline in Joint Range of Motion.Baseline, 25 weeks, 52 weeks
SecondaryChange From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.Baseline to 25 weeks
SecondaryChange From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.Baseline, 25 weeks, 52 weeks
SecondaryChange from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)25, 52 weeks
SecondaryChange from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )25, 52 weeks
SecondaryDrug concentration in Cerebrospinal Fluid.25, 52*weeks\*Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .

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/NCT03568175/registered