Ignite Creation Date:
2024-05-06 @ 2:06 PM
Last Modification Date:
2024-10-26 @ 1:25 PM
Study NCT ID:
NCT04219007
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-02-06
First Post:
2019-12-26
Brief Title:
Vosoritide for Selected Genetic Causes of Short Stature
Sponsor:
Andrew Dauber
Organization:
Childrens National Research Institute
Study Overview
Official Title:
Vosoritide for Selected Genetic Causes of Short Stature
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-02
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:
Short stature can be caused by a number of genetic etiologies many of which directly affect the growth plate The FGFR3CNP pathway is central to growth of the chondrocyte The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide a CNP analog a selective NPR-B agonist which directly targets the growth plate This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity safety profile and quality of life assessment Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes
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