Ignite Creation Date:
2025-12-25 @ 3:20 AM
Ignite Modification Date:
2026-03-02 @ 3:44 PM
Study NCT ID:
NCT03651505
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-31
First Post:
2018-08-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
X-linked Hypophosphatemia Disease Monitoring Program
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Study Overview
Official Title:
X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
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:
None
Brief Summary:
The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
Detailed Description:
The XLH-DMP is a global, prospective, multicenter, longitudinal, long-term outcomes program for subjects on or off any treatment designed to characterize XLH disease presentation and progression, assess long-term safety and effectiveness of burosumab, as well as prospectively investigate longitudinal change over time across biomarker(s), clinical assessments, and patient/caregiver-reported outcome measures in a representative population. The XLH-DMP will collect demographic, biochemical, physiologic, disease severity, and progression data in patients taking burosumab and those not taking burosumab. In this DMP, patients will only have access to burosumab through authorized prescribed use. The Sponsor will not provide any treatments as part of the DMP.
Study Oversight
Has Oversight DMC:
False
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?: