X-linked Hypophosphatemia Disease Monitoring Program

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


Lead Sponsor: Ultragenyx Pharmaceutical Inc

Collaborator: Kyowa Kirin Co., Ltd.

Source 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.

Overall Status Recruiting
Start Date 2018-07-16
Completion Date 2028-07-01
Primary Completion Date 2028-07-01
Study Type Observational
Primary Outcome
Measure Time Frame
Long-Term Safety of Burosumab 10 years
Long-Term Effectiveness of Burosumab 10 years
Clinical Course of XLH Disease 10 years
Enrollment 800

Intervention Type: Other

Intervention Name: No intervention

Description: Access to any treatment is through authorized commercial use and not as a part of this DMP


Sampling Method:

Non-Probability Sample


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



Minimum Age:


Maximum Age:


Healthy Volunteers:


Overall Official
Last Name Role Affiliation
Ultragenyx Medical Director Study Director Ultragenyx Pharmaceuticals Inc.
Overall Contact

Last Name: Patients Contact: Patient Advocacy

Phone: 1-415-483-8800

Email: [email protected]

Facility: Status:
Children's Hospital of Alabama | Birmingham, Alabama, 35233, United States Recruiting
Children's Hospital Los Angeles | Los Angeles, California, 90027, United States Recruiting
Rady Children's Hospital San Diego | San Diego, California, 92123, United States Recruiting
University of California San Francisco | San Francisco, California, 94158, United States Active, not recruiting
Children's Hospital of Colorado | Aurora, Colorado, 80045, United States Recruiting
Yale University School of Medicine | New Haven, Connecticut, 06520, United States Active, not recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, Illinois, 60611, United States Active, not recruiting
Indiana University School of Medicine | Indianapolis, Indiana, 46202-5167, United States Active, not recruiting
Johns Hopkins University | Baltimore, Maryland, 21218, United States Recruiting
Boston Children's Hospital | Boston, Massachusetts, 02115, United States Recruiting
Duke University | Durham, North Carolina, 27708, United States Active, not recruiting
Cincinnati Children's Hospital | Cincinnati, Ohio, 45229, United States Recruiting
Nationwide Children's Hospital | Columbus, Ohio, 43205, United States Active, not recruiting
The Ohio State University Wexner Medical Center | Columbus, Ohio, 43205, United States Active, not recruiting
Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, 19104, United States Recruiting
Vanderbilt University - Department of Medicine | Nashville, Tennessee, 37212, United States Recruiting
Vanderbilt University - Department of Pediatrics | Nashville, Tennessee, 37232, United States Recruiting
Houston Methodist Research Institute | Houston, Texas, 77030, United States Active, not recruiting
University of Texas, Health Science Center | Houston, Texas, 77030, United States Active, not recruiting
Children's Hospital of The King's Daughter | Norfolk, Virginia, 23507, United States Recruiting
Seattle Children's Hospital | Seattle, Washington, 98105, United States Recruiting
American Family Children's Hospital | Madison, Wisconsin, 53792, United States Recruiting
Hospital General de Niños Pedro de Elizalde | Buenos Aires, Ciudad Autónoma De Buenos Aires, C1270AAN, Argentina Recruiting
Hospital de Niños Dr. Ricardo Gutierrez | Buenos Aires, C1425EFD, Argentina Recruiting
Hospital Universitário Alcides Carneiro (HUAC) | São José, Campina Grande - PB, 58400-398, Brazil Active, not recruiting
Serviço de Endocrinologia e Metabologia - Hospital de Clínicas da Universidade Federal do Para (SEMPR) | Curitiba, Parana, 80030-110, Brazil Recruiting
Instituto de Medicina Avancada (IMA Brasil) | São Paulo, Sao Paulo, 05437-000, Brazil Active, not recruiting
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo | São Paulo, 05403-000, Brazil Recruiting
British Columbia Children's Hospital | Vancouver, British Columbia, V6H 3N1, Canada Recruiting
Bone Research and Education Centre | Oakville, Ontario, ON L6M 1M1, Canada Recruiting
Shiner's Hospital for Children - Canada | Montréal, Quebec, H4A 0A9, Canada Recruiting
Children's Hospital Eastern Ontario Research Institute | Ottawa, K1H 5B2, Canada Recruiting
Pontificia Universidad Católica de Chile | Santiago, Metropolitana, Chile Recruiting
Hospital Universitario de San Vicente Fundación | Medellín, Antioquia, Colombia Recruiting
Hospital Infantil Universitario de San José | Bogotá, Cundinamarca, Colombia Recruiting
Location Countries






United States

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Arm Group

Label: Prior Burosumab Clinical Trial Participants

Description: Patients who participated in burosumab clinical trials and continue to receive burosumab via prescription from their physician.

Label: Not from Prior Burosumab Clinical Trial

Description: Patients may take other treatments for XLH and may start burosumab treatment at any time as prescribed by a physician.

Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

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