Ignite Creation Date:
2024-05-06 @ 6:58 PM
Last Modification Date:
2024-10-26 @ 2:58 PM
Study NCT ID:
NCT05849389
Status:
RECRUITING
Last Update Posted:
2024-06-24
First Post:
2023-04-28
Brief Title:
Vosoritide for Short Stature in Turner Syndrome
Sponsor:
Roopa Kanakatti Shankar MBBS MS
Organization:
Childrens National Research Institute
Study Overview
Official Title:
Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None