protocolSection identificationModule nctId: NCT06382155, orgStudyIdInfo id: 111-210, briefTitle: A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature, statusModule overallStatus: NOT_YET_RECRUITING, startDateStruct date: 2024-09, primaryCompletionDateStruct date: 2026-06, completionDateStruct date: 2036-12, studyFirstPostDateStruct date: 2024-04-24, lastUpdatePostDateStruct date: 2024-04-24, sponsorCollaboratorsModule leadSponsor name: BioMarin Pharmaceutical, class: INDUSTRY, descriptionModule briefSummary: The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH), in children with idiopathic short stature (ISS)., conditionsModule conditions: Idiopathic Short Stature, designModule studyType: INTERVENTIONAL, phases: PHASE2, designInfo allocation: RANDOMIZED, interventionModel: PARALLEL, primaryPurpose: TREATMENT, maskingInfo masking: QUADRUPLE, whoMasked: PARTICIPANT, whoMasked: CARE_PROVIDER, whoMasked: INVESTIGATOR, whoMasked: OUTCOMES_ASSESSOR, enrollmentInfo count: 100, type: ESTIMATED, armsInterventionsModule interventions name: Vosoritide Injection, interventions name: Human Growth Hormone, interventions name: Placebo, outcomesModule primaryOutcomes measure: Change from baseline in Annualized Growth Velocity (AGV), primaryOutcomes measure: Change from baseline in height, primaryOutcomes measure: Change from baseline in height Z-score, secondaryOutcomes measure: Incidence of treatment-emergent adverse events, secondaryOutcomes measure: Change from baseline in AGV Z-score (average stature reference), secondaryOutcomes measure: Change from baseline in height, secondaryOutcomes measure: Change from baseline in height Z score, secondaryOutcomes measure: Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP), secondaryOutcomes measure: Change from baseline at pre-specified timepoints in serum collagen X marker (CXM), secondaryOutcomes measure: Change from baseline in bone age minus chronological age at pre-specified timepoints, secondaryOutcomes measure: Change from baseline in total body (less head) bone mineral density (BMD) Z-score, secondaryOutcomes measure: Change from baseline in lumbar spine BMD Z-score, secondaryOutcomes measure: Change from baseline in total body (less head) bone mineral content (BMC), secondaryOutcomes measure: Change from baseline in lumbar spine BMC, secondaryOutcomes measure: Maximum concentration (Cmax) of vosoritide in plasma, secondaryOutcomes measure: Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞), secondaryOutcomes measure: Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t), secondaryOutcomes measure: Elimination half-life of vosoritide (t½), secondaryOutcomes measure: Apparent clearance of vosoritide, secondaryOutcomes measure: Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F), secondaryOutcomes measure: Time vosoritide is present at maximum concentration (Tmax), eligibilityModule sex: ALL, minimumAge: 3 Years, maximumAge: 10 Years, stdAges: CHILD, hasResults: False