Ignite Creation Date:
2025-12-24 @ 2:58 PM
Ignite Modification Date:
2025-12-27 @ 2:05 PM
Study NCT ID:
NCT06455059
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-14
First Post:
2024-06-07
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Sponsor:
BioMarin Pharmaceutical
Organization:
Study Overview
Official Title:
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-10
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:
CANOPY-HCH-3
Brief Summary:
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
Detailed Description:
This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.
Study Oversight
Has Oversight DMC:
True
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?:
False
Is an FDA AA801 Violation?: