Trial · NCT07073014
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT07073014, Phase 3, Enrolling by invitation, BioMarin Pharmaceutical
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia. Trials, record NCT07073014. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07073014
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
- NCT number
- NCT07073014
- Status
- Enrolling by invitation
- Phases
- Phase 3
- Enrollment
- 140 participants, estimated
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Hypochondroplasia
- Interventions
- Vosoritide
- First posted
- 2025-07-18
- Last updated
- 2026-09-09
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Evaluate 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 males | As measured by change from baseline in height Z-score |
| Secondary | Evaluate the maintenance effect of vosoritide treatment on standing height | Baseline to week 52 | change from baseline in height at Week 52 |
| Secondary | Evaluate the maintenance effect of vosoritide treatment on standing height Z-score | Baseline to week 52 | Change from baseline in height Z-score at Week 52 |
| Secondary | Evaluate long-term effect of vosoritide treatment on growth velocity | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males | Absolute 6- or 12-month interval AGV |
| Secondary | Evaluate long-term effect of vosoritide treatment on body proportionality | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males | Change from baseline in upper to lower body segment ratio |
| Secondary | Evaluate long-term effect of vosoritide treatment on bone age versus chronological age | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males | As measured by left-hand and wrist x-ray |
| Secondary | Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males | Change from baseline in serum collagen X marker |
| Secondary | Evaluate 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 males | Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score |
| Secondary | Evaluate long-term effect of vosoritide treatment on bone quality | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males | Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry |
| Secondary | Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging | Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
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.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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.
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/NCT07073014
GET https://conductscience.org/api/trials/v1/NCT07073014/registered