X-linked Hypophosphatemia Disease Monitoring Program

Running, not enrolling

Conditions studied: X-linked Hypophosphatemia, Hypophosphatemic Rickets

In brief

The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.

Key facts

Study ID

NCT03651505

Run by

Ultragenyx Pharmaceutical Inc

People needed

782

Starts

2018-07-16

Expected to finish

2032-12-01

Last updated by the study team

2026-05-22

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Concurrent enrollment in an Ultragenyx-sponsored clinical trial is NOT permitted.
• Serious medical or psychiatric comorbidity.
• Less than one year of life expectancy.

Where it is running

• 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
• Washington University School of Medicine — St Louis, Missouri, United States
• Duke University — Durham, North Carolina, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• The Ohio State University Wexner Medical Center — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Vanderbilt University - Department of Medicine — Nashville, Tennessee, United States
• Vanderbilt University - Department of Pediatrics — Nashville, Tennessee, United States
• Houston Methodist Research Institute — Houston, Texas, United States
• University of Texas, Health Science Center — Houston, Texas, United States
• University of Virgina School of Medicine — Charlottesville, Virginia, United States
• Children's Hospital of The King's Daughter — Norfolk, Virginia, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• American Family Children's Hospital — Madison, Wisconsin, United States
• Hospital General de Niños Pedro de Elizalde — Buenos Aires, Buenos Aires F.D., Argentina

Full record on ClinicalTrials.gov

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