X-linked Hypophosphatemia Disease Monitoring Program
Phase: Recruiting
NCT Number: NCT03651505 Other Study ID Number(s): UX023-CL401
What Is the Purpose of This Study?
X-linked hypophosphatemia (XLH), is a rare genetic disorder caused by mutations in the PHEX gene that leads to poor bone health, bone pain, and fractures. The X-linked Hypophosphatemia Disease Monitoring Program (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 (Ultragenyx), 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.
For a full list of eligibility requirements, visit https://clinicaltrials.gov/study/NCT03651505