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Trial · NCT04219007

Vosoritide for Selected Genetic Causes of Short Stature

NCT04219007, Phase 2, Active not recruiting, Andrew Dauber

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Vosoritide for Selected Genetic Causes of Short Stature. Trials, record NCT04219007. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04219007

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title
Vosoritide for Selected Genetic Causes of Short Stature
NCT number
NCT04219007
Status
Active not recruiting
Phases
Phase 2
Enrollment
56 participants, actual
Lead sponsor
Andrew Dauber
Conditions
Short Stature
Interventions
Vosoritide
First posted
2020-01-06
Last updated
2026-03-18

Source ClinicalTrials.gov

Registered outcomes16

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

ListOutcomeTime frameDescription
PrimaryIncidence of Treatment Emergent Adverse Events [Safety and Tolerability]12 monthsNumber of treatment-emergent adverse events or serious adverse events per study participant
PrimaryIncidence of Symptomatic Hypotension events [Safety and Tolerability]12 monthsNumber of symptomatic hypotension events per study participant
PrimaryChange from Baseline in annualized growth velocity12 months
To evaluate the change from baseline in age-sex standardized annualized growth velocity in cm/year after 12 months of daily SC injections of vosoritide in patients with selected genetic causes of short stature.
PrimaryChange from Baseline in standardized height SDS12 monthsTo evaluate the change from baseline in age-sex standardized height SDS after 12 months of daily SC injections of vosoritide in patients with selected genetic causes of short stature.
SecondaryChanges in the seated height ratio as a measure of body proportions12 monthsTo evaluate the seated height ratio (expressed as a percentage) as a measure of body proportions. Measurement after 12 months of treatment will be compared to baseline.
SecondaryChanges in the difference between arm span and height from baseline12 monthsTo evaluate the difference between arm span and height (measured as cm) as a measure of body proportions. Measurement after 12 months of treatment will be compared to baseline.
SecondaryChanges from Baseline in bone age/chronological age12 monthsTo evaluate changes from baseline in bone age/chronological age after 12 months of daily SC injections of vosoritide
OtherCharacterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)3 hours at each visitEstimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant
OtherCharacterize the area under the plasma concentration-time curve from 0 to the time of last measurable concentration (AUC0-t)3 hours at each visitEstimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant
OtherCharacterize maximum concentration (Cmax) of vosoritide in plasma3 hours at each visitEstimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant

Outcomes0

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

None in this release.

Results by group0

Each measurement the trial posted, group by group, in the words and numbers it posted.

None in this release.

Participant flow0

How many started, completed or left each group, period by period.

None in this release.

Adverse events0

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

None in this release.

Adverse event totals0

The totals the trial posted for each group.

None in this release.

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMIDListed as
38813446DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov
2026-11-12
ClinicalTrials.gov
Results, from AACT
2026-03-23
AACT
NIH grant links
2026-11-13
NIH RePORTER

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.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04219007

GET https://conductscience.org/api/trials/v1/NCT04219007/registered