X-linked Hypophosphatemia Disease Monitoring Program
- Conditions
- Hypophosphatemic RicketsX-linked Hypophosphatemia
- Registration Number
- NCT03651505
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 782
- Willing and able to provide informed consent or, in the case of patients under the age of 18 years (or 16 years, depending on the region), provide assent (if required) and informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- Clinical diagnosis of XLH based on family history, OR confirmed PHEX mutation, OR biochemical profile consistent with XLH.
- Willing and able to comply with the study visit schedule and study procedures.
- Concurrent enrollment in an Ultragenyx-sponsored clinical trial is NOT permitted.
- Serious medical or psychiatric comorbidity.
- Less than one year of life expectancy.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Long-Term Safety of Burosumab 10 years 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.
Long-Term Effectiveness of Burosumab 10 years To evaluate the long-term effectiveness of burosumab treatment on key manifestations of XLH, including skeletal health, stiffness, mobility and physical functioning.
Clinical Course of XLH Disease 10 years To illustrate the clinical, radiological, biochemical manifestations and progression of XLH over time in both untreated and treated patients with XLH.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (38)
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
Scroll for more (28 remaining)Children's Hospital of Alabama🇺🇸Birmingham, Alabama, United States