Trial · NCT04219007
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 16 of 16
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence of Treatment Emergent Adverse Events [Safety and Tolerability] | 12 months | Number of treatment-emergent adverse events or serious adverse events per study participant |
| Primary | Incidence of Symptomatic Hypotension events [Safety and Tolerability] | 12 months | Number of symptomatic hypotension events per study participant |
| Primary | Change from Baseline in annualized growth velocity | 12 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. |
| Primary | Change from Baseline in standardized height SDS | 12 months | To 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. |
| Secondary | Changes in the seated height ratio as a measure of body proportions | 12 months | To 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. |
| Secondary | Changes in the difference between arm span and height from baseline | 12 months | To 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. |
| Secondary | Changes from Baseline in bone age/chronological age | 12 months | To evaluate changes from baseline in bone age/chronological age after 12 months of daily SC injections of vosoritide |
| Other | Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞) | 3 hours at each visit | Estimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant |
| Other | Characterize the area under the plasma concentration-time curve from 0 to the time of last measurable concentration (AUC0-t) | 3 hours at each visit | Estimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant |
| Other | Characterize maximum concentration (Cmax) of vosoritide in plasma | 3 hours at each visit | Estimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant |
| Other | Characterize the elimination half-life of vosoritide (t½) | 3 hours at each visit | Estimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant |
| Other | Characterize the apparent clearance of drug (CL/F) | 3 hours at each visit | Estimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant |
| Other | Characterize the apparent volume of distribution based upon the terminal phase (Vz/F) | 3 hours at each visit | Estimated by non-compartmental analysis for Day 1, 6 months, and 12 months per study participant |
| Other | Changes from Baseline in bone mineral density | 12 months | To evaluate changes from baseline in bone mineral density (BMD) after 12 months of daily SC injections of vosoritide (expressed as height adjusted Z-score). |
| Other | Evaluate immunogenicity and assess impact on safety and efficacy measures | 12 months | To evaluate immunogenicity and assess impact on safety and efficacy measures after 12 months of daily SC injections of vosoritide as evidenced by rates of vosoritide antibody development, which is a standard safety metric. |
| Other | Changes from baseline in quality of life after daily SC injections of vosoritide: Quality of Life of Short Stature Youth (QoLISSY) questionnaire score is expressed as a percentage | 12 months | To evaluate changes from baseline in quality of life using the QoLISSY scale after 12 months of daily SC injections of vosoritide. The Quality of Life of Short Stature Youth (QoLISSY) questionnaire score is expressed as a percentage, with scores ranging from 0% to 100% (100% is the best score). |
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.
For software
Read this list from software.
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GET https://conductscience.org/api/trials/v1/NCT04219007/registered