Trial · Interventional
Status unknownPhase 2Registry dataVosoritide for Short Stature in Turner Syndrome
Overview
- Status
- Status unknown
- Phase
- Phase 2
- Enrollment
- 20 participants (estimated)
- Sponsor
- Roopa Kanakatti Shankar, MBBS, MS · Other
- Design
- Allocation not applicable · Open label (no masking)
- Countries
1 country
United States
- Start
- 12 Apr 2024
- Primary completion
- Sept 2026 (estimated)
- Completion
- Sept 2026 (estimated)
- First posted
- 8 May 2023
- Last update posted
- 24 Jun 2024
- Conditions
- Turner Syndrome · Short Stature
Timeline
1 trial
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
- Ph 2
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
Turner syndrome (TS) is characterized by a missing whole or part of the second sex chromosome in a phenotypic female, resulting in short stature due to haploinsufficiency of the short-stature homeobox-containing (SHOX) gene. Growth hormone (GH) is an approved therapy for this condition, although not associated with GH deficiency, and benefits are modest. Vosoritide, a C-type natriuretic peptide (CNP) analog, targets chondrocytes within the growth plate leading to increased cell proliferation and hypertrophy. We hypothesize that patients with TS and short stature will respond to vosoritide treatment leading to increased growth velocity. This study will enroll pre-pubertal girls with TS who are either naïve to GH or have had a poor response to GH therapy. All subjects will be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes. Annualized growth velocity (AGV) on vosoritide will be compared to AGV in the 6-18 months prior to initiation of vosoritide based on historical data available in the medical record. Subjects with a positive response to therapy will be given the option to continue in the extension phase of the study during which they will continue to receive vosoritide until growth cessation.
Interventions
- VosoritideDrug
Arms1
Experimental
Vosoritide treatment arm
Vosoritide will be administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia.
Primary outcomes3
Outcomes the sponsor listed as primary.
Incidence of treatment-emergent adverse events
Time frame · 12 months
Registry description
Number of treatment-emergent adverse events or serious adverse events per study participant
Change from baseline in annualized growth velocity
Time frame · 12 months
Registry description
To evaluate the change from baseline in annualized growth velocity after 12 months of daily subcutaneous injections of vosoritide
Change from baseline in age-sex standardized height standard deviation score
Time frame · 12 months
Registry description
To evaluate the change from baseline in age-sex standardized height standard deviation score (SDS) after 12 months of daily subcutaneous injections of vosoritide
Secondary outcomes(3)
Changes in seated height ratio
Time frame · 12 months
Registry description
To evaluate the seated height ratio as a measure of body proportions compared to baseline after daily subcutaneous injections of vosoritide
Changes in arm span minus standing height
Time frame · 12 months
Registry description
To evaluate the arm span minus standing height as a measure of body proportion compared to baseline after daily subcutaneous injections of vosoritide
Change in Bone Age
Time frame · 12 months
Registry description
To evaluate changes from baseline in bone age/chronological age after 12 months of daily subcutaneous injections of vosoritide
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
No results are posted for this trial.
A trial that is still running, or that stopped early, may post results later.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- Female
- Age
- from 3 Years up to 11 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
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