X-linked Hypophosphatemia Disease Monitoring Program

March 7, 2024 updated by: Ultragenyx Pharmaceutical Inc

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

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

780

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina, C1425EFD
        • Hospital de Niños Dr. Ricardo Gutiérrez
      • Córdoba, Argentina
        • Hospital Privado Universitario de Córdoba
    • Ciudad Autónoma De Buenos Aires
      • Buenos Aires, Ciudad Autónoma De Buenos Aires, Argentina, C1270AAN
        • Hospital General de Niños Pedro de Elizalde
  • Brazil
      • Curitiba, Brazil, 80030-110
        • Ceti - Centro de Estudos Em Terapias Inovadoras
      • São Paulo, Brazil, 05403-000
        • Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo
    • Campina Grande - PB
      • São José, Campina Grande - PB, Brazil, 58400-398
        • Hospital Universitário Alcides Carneiro (HUAC)
    • Sao Paulo
      • São Paulo, Sao Paulo, Brazil, 05437-000
        • Instituto de Medicina Avancada (IMA Brasil)
  • Canada
      • Ottawa, Canada, K1H 5B2
        • Children's Hospital Eastern Ontario Research Institute
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3N1
        • British Columbia Children's Hospital
    • Ontario
      • Oakville, Ontario, Canada, ON L6M 1M1
        • Bone Research and Education Centre
    • Quebec
      • Montréal, Quebec, Canada, H4A 0A9
        • Shiner's Hospital for Children - Canada
  • Chile
    • Metropolitana
      • Santiago, Metropolitana, Chile
        • Pontificia Universidad Catolica de Chile
  • Colombia
    • Antioquia
      • Medellín, Antioquia, Colombia
        • Hospital Universitario de San Vicente Fundación
    • Cundinamarca
      • Bogotá, Cundinamarca, Colombia
        • Hospital Infantil Universitario de San José
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Children's Hospital of Alabama
    • California
      • Los Angeles, California, United States, 90095
        • University of California, Los Angeles
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • San Diego, California, United States, 92123
        • Rady Children's Hospital San Diego
      • San Francisco, California, United States, 94158
        • University of California San Francisco
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital of Colorado
    • Connecticut
      • New Haven, Connecticut, United States, 06520
        • Yale University School of Medicine
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202-5167
        • Indiana University School of Medicine
    • Maryland
      • Baltimore, Maryland, United States, 21218
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
      • Boston, Massachusetts, United States, 02115
        • Karp Family Research Laboratories
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • North Carolina
      • Durham, North Carolina, United States, 27708
        • Duke University
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
      • Columbus, Ohio, United States, 43205
        • The Ohio State University Wexner Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Vanderbilt University - Department of Medicine
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University - Department of Pediatrics
    • Texas
      • Houston, Texas, United States, 77030
        • Houston Methodist Research Institute
      • Houston, Texas, United States, 77030
        • University of Texas, Health Science Center
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughter
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • American Family Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Approximately 800 eligible adult and pediatric patients with XLH will be enrolled globally, with a minimum of 200 pediatric patients. Patients can enter the XLH-DMP regardless of how their XLH is being treated. Patients on Crysvita (burosumab) via prescription may begin taking Crysvita, per standard of care before or after enrolling in the XLH DMP.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Prior Burosumab Clinical Trial Participants
Patients who participated in burosumab clinical trials and continue to receive burosumab via prescription from their physician.
Other: No intervention
Access to any treatment is through authorized commercial use and not as a part of this DMP
Not from Prior Burosumab Clinical Trial
Patients may take other treatments for XLH and may start burosumab treatment at any time as prescribed by a physician.
Other: No intervention
Access to any treatment is through authorized commercial use and not as a part of this DMP

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Long-Term Safety of Burosumab
Time Frame: 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.
10 years
Long-Term Effectiveness of Burosumab
Time Frame: 10 years
To evaluate the long-term effectiveness of burosumab treatment on key manifestations of XLH, including skeletal health, stiffness, mobility and physical functioning.
10 years
Clinical Course of XLH Disease
Time Frame: 10 years
To illustrate the clinical, radiological, biochemical manifestations and progression of XLH over time in both untreated and treated patients with XLH.
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ultragenyx Pharmaceutical Inc

Collaborators

Kyowa Kirin Co., Ltd.

Investigators

  • Study Director: Ultragenyx Medical Director, Ultragenyx Pharmaceuticals Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 16, 2018

Primary Completion (Estimated)

December 1, 2032

Study Completion (Estimated)

December 1, 2032

Study Registration Dates

First Submitted

August 24, 2018

First Submitted That Met QC Criteria

August 28, 2018

First Posted (Actual)

August 29, 2018

Study Record Updates

Last Update Posted (Actual)

March 8, 2024

Last Update Submitted That Met QC Criteria

March 7, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UX023-CL401

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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