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XLH study: watching the disease, not curing it

NCT ID NCT03651505

Knowledge-focused Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 1 time

Summary

This observational study follows 782 people with X-linked hypophosphatemia (XLH), a rare genetic bone disease, to understand how the condition changes over time. It also monitors the long-term safety and effectiveness of the drug burosumab. No new treatment is given; participants continue their usual care.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

782 people

The number who actually took part.

Started

Jul 2018

Expected to finish

Dec 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

hypophosphatemia hypophosphatemic rickets X-linked dominant hypophosphatemic rickets X-linked hypophosphatemic rickets

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hypophosphatemic rickets X-linked hypophosphatemia

Contacts and locations

Locations

  • American Family Children's Hospital

    Madison, Wisconsin, 53792, United States

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Bone Research and Education Centre

    Oakville, Ontario, ON L6M 1M1, Canada

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3N1, Canada

  • CETI - Centro de Estudos em Terapias Inovadoras

    Curitiba, 80030-110, Brazil

  • Children's Hospital Eastern Ontario Research Institute

    Ottawa, K1H 5B2, Canada

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Alabama

    Birmingham, Alabama, 35233, United States

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of The King's Daughter

    Norfolk, Virginia, 23507, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Duke University

    Durham, North Carolina, 27708, United States

  • Hospital General de Niños Pedro de Elizalde

    Buenos Aires, Buenos Aires F.D., C1270AAN, Argentina

  • Hospital Infantil Universitario de San José

    Bogotá, Cundinamarca, Colombia

  • Hospital Privado Universitario de Córdoba

    Córdoba, Argentina

  • Hospital Universitario de San Vicente Fundación

    Medellín, Antioquia, Colombia

  • Hospital Universitário Alcides Carneiro (HUAC)

    São José, Campina Grande - PB, 58400-398, Brazil

  • Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

    São Paulo, São Paulo, 05403-000, Brazil

  • Hospital de Niños Dr. Ricardo Gutierrez

    Buenos Aires, Buenos Aires, C1425EFD, Argentina

  • Houston Methodist Research Institute

    Houston, Texas, 77030, United States

  • Indiana University School of Medicine

    Indianapolis, Indiana, 46202-5167, United States

  • Instituto de Medicina Avancada (IMA Brasil)

    São Paulo, São Paulo, 05437-000, Brazil

  • Johns Hopkins University

    Baltimore, Maryland, 21218, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Pontificia Universidad Católica de Chile

    Santiago, Santiago Metropolitan, Chile

  • Rady Children's Hospital San Diego

    San Diego, California, 92123, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Shiner's Hospital for Children - Canada

    Montreal, Quebec, H4A 0A9, Canada

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43205, United States

  • University of California San Francisco

    San Francisco, California, 94158, United States

  • University of Texas, Health Science Center

    Houston, Texas, 77030, United States

  • University of Virgina School of Medicine

    Charlottesville, Virginia, 22903, United States

  • Vanderbilt University - Department of Medicine

    Nashville, Tennessee, 37212, United States

  • Vanderbilt University - Department of Pediatrics

    Nashville, Tennessee, 37232, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Yale University School of Medicine

    New Haven, Connecticut, 06520, United States

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