The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
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 Type
OBSERVATIONAL
Enrollment
782
Access to any treatment is through authorized commercial use and not as a part of this DMP
Long-Term Safety of Burosumab
To assess the long-term safety of burosumab treatment in adult and pediatric patients with XLH, including overall renal health, the presence and/or progression of nephrocalcinosis and spinal stenosis, and pregnancy outcomes.
Time frame: 10 years
Long-Term Effectiveness of Burosumab
To evaluate the long-term effectiveness of burosumab treatment on key manifestations of XLH, including skeletal health, stiffness, mobility and physical functioning.
Time frame: 10 years
Clinical Course of XLH Disease
To illustrate the clinical, radiological, biochemical manifestations and progression of XLH over time in both untreated and treated patients with XLH.
Time frame: 10 years
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Children's Hospital of Alabama
Birmingham, Alabama, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Rady Children's Hospital San Diego
San Diego, California, United States
University of California San Francisco
San Francisco, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
Yale University School of Medicine
New Haven, Connecticut, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Johns Hopkins University
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
...and 28 more locations