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Trial · NCT07073014

Registered outcomes12

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

ListOutcomeTime frameDescription
PrimaryEvaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesAs measured by change from baseline in height Z-score
SecondaryEvaluate the maintenance effect of vosoritide treatment on standing heightBaseline to week 52change from baseline in height at Week 52
SecondaryEvaluate the maintenance effect of vosoritide treatment on standing height Z-scoreBaseline to week 52Change from baseline in height Z-score at Week 52
SecondaryEvaluate long-term effect of vosoritide treatment on growth velocityAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesAbsolute 6- or 12-month interval AGV
SecondaryEvaluate long-term effect of vosoritide treatment on body proportionalityAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesChange from baseline in upper to lower body segment ratio
SecondaryEvaluate long-term effect of vosoritide treatment on bone age versus chronological ageAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesAs measured by left-hand and wrist x-ray
SecondaryEvaluate long-term effect of vosoritide treatment on bone metabolism biomarkersAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesChange from baseline in serum collagen X marker
SecondaryEvaluate long-term changes in Health-Related Quality of Life (HRQoL)Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesChange from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score
SecondaryEvaluate long-term effect of vosoritide treatment on bone qualityAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for malesChange from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry
SecondaryEvaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner StagingAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
SecondaryTo monitor the incidence of otitis mediaAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
SecondaryTo monitor the frequency of seizuresAnnually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

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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