X-linked Hypophosphatemia Disease Monitoring Program

NCT03651505 · tracked via the Priya Life Science USA tracker

Sponsor

Ultragenyx Pharmaceutical Inc

Phase

Observational

Started

2018-07-16

Last updated

2026-08-28

Condition(s) studied

X-linked HypophosphatemiaHypophosphatemic Rickets

Investigational drug(s) / intervention(s)

No intervention

No intervention: Access to any treatment is through authorized commercial use and not as a part of this DMP

Study 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.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

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.

Trial sites (38)

Facility	City	Region
Children's Hospital of Alabama	Birmingham	Alabama
Children's Hospital Los Angeles	Los Angeles	California
Rady Children's Hospital San Diego	San Diego	California
University of California San Francisco	San Francisco	California
Children's Hospital of Colorado	Aurora	Colorado
Yale University School of Medicine	New Haven	Connecticut
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
Indiana University School of Medicine	Indianapolis	Indiana
Johns Hopkins University	Baltimore	Maryland
Boston Children's Hospital	Boston	Massachusetts
Washington University School of Medicine	St Louis	Missouri
Duke University	Durham	North Carolina
Cincinnati Children's Hospital	Cincinnati	Ohio
Nationwide Children's Hospital	Columbus	Ohio
The Ohio State University Wexner Medical Center	Columbus	Ohio
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
Vanderbilt University - Department of Medicine	Nashville	Tennessee
Vanderbilt University - Department of Pediatrics	Nashville	Tennessee
Houston Methodist Research Institute	Houston	Texas
University of Texas, Health Science Center	Houston	Texas
University of Virgina School of Medicine	Charlottesville	Virginia
Children's Hospital of The King's Daughter	Norfolk	Virginia
Seattle Children's Hospital	Seattle	Washington
American Family Children's Hospital	Madison	Wisconsin
Hospital de Niños Dr. Ricardo Gutierrez	Buenos Aires	Buenos Aires
Hospital General de Niños Pedro de Elizalde	Buenos Aires	Buenos Aires F.D.
Hospital Privado Universitario de Córdoba	Córdoba	Argentina
Hospital Universitário Alcides Carneiro (HUAC)	São José	Campina Grande - PB
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo	São Paulo	São Paulo
Instituto de Medicina Avancada (IMA Brasil)	São Paulo	São Paulo
CETI - Centro de Estudos em Terapias Inovadoras	Curitiba	Brazil
British Columbia Children's Hospital	Vancouver	British Columbia
Bone Research and Education Centre	Oakville	Ontario
Shiner's Hospital for Children - Canada	Montreal	Quebec
Children's Hospital Eastern Ontario Research Institute	Ottawa	Canada
Pontificia Universidad Católica de Chile	Santiago	Santiago Metropolitan
Hospital Universitario de San Vicente Fundación	Medellín	Antioquia
Hospital Infantil Universitario de San José	Bogotá	Cundinamarca

More Ultragenyx Pharmaceutical Inc trials in the USA

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) Active, not recruiting

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) Active, not recruiting

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency Active, not recruiting

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency Active, not recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH Recruiting

Other trials for the same condition

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03651505 on ClinicalTrials.gov ↗ ← All trials in the USA