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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Vosoritide administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia.
Children's National Hospital
Washington D.C., District of Columbia, United States
RECRUITINGIncidence of treatment-emergent adverse events
Number of treatment-emergent adverse events or serious adverse events per study participant
Time frame: 12 months
Change from baseline in annualized growth velocity
To evaluate the change from baseline in annualized growth velocity after 12 months of daily subcutaneous injections of vosoritide
Time frame: 12 months
Change from baseline in age-sex standardized height standard deviation score
To evaluate the change from baseline in age-sex standardized height standard deviation score (SDS) after 12 months of daily subcutaneous injections of vosoritide
Time frame: 12 months
Changes in seated height ratio
To evaluate the seated height ratio as a measure of body proportions compared to baseline after daily subcutaneous injections of vosoritide
Time frame: 12 months
Changes in arm span minus standing height
To evaluate the arm span minus standing height as a measure of body proportion compared to baseline after daily subcutaneous injections of vosoritide
Time frame: 12 months
Change in Bone Age
To evaluate changes from baseline in bone age/chronological age after 12 months of daily subcutaneous injections of vosoritide
Time frame: 12 months
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