NCT03651505

X-linked Hypophosphatemia Disease Monitoring Program

X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 782 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• X-linked Hypophosphatemia
• Hypophosphatemic Rickets

Interventions

Type	Name	Description
OTHER	No intervention	Access to any treatment is through authorized commercial use and not as a part of this DMP

Timeline

• Start date: 2018-07-16
• Primary completion: 2032-12-01
• Completion: 2032-12-01
• First posted: 2018-08-29
• Last updated: 2026-02-17

Locations

38 sites across 6 countries: United States, Argentina, Brazil, Canada, Chile, Colombia

Regulatory

• FDA-regulated drug study