X-linked Hypophosphatemia Disease Monitoring Program

NCT ID: NCT03651505

Last Updated: 2025-10-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

782 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-07-16

Study Completion Date

2032-12-31

Brief Summary

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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

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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.

Conditions

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X-linked Hypophosphatemia Hypophosphatemic Rickets

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Prior Burosumab Clinical Trial Participants

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

No intervention

Intervention Type OTHER

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.

No intervention

Intervention Type OTHER

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

Interventions

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No intervention

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

Intervention Type OTHER

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Kyowa Kirin Co., Ltd.

INDUSTRY

Sponsor Role collaborator

Ultragenyx Pharmaceutical Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ultragenyx Medical Director

Role: STUDY_DIRECTOR

Ultragenyx Pharmaceuticals Inc.

Locations

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Children's Hospital of Alabama

Birmingham, Alabama, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Rady Children's Hospital San Diego

San Diego, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

Children's Hospital of Colorado

Aurora, Colorado, United States

Site Status

Yale University School of Medicine

New Haven, Connecticut, United States

Site Status

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

Chicago, Illinois, United States

Site Status

Indiana University School of Medicine

Indianapolis, Indiana, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University - Department of Medicine

Nashville, Tennessee, United States

Site Status

Vanderbilt University - Department of Pediatrics

Nashville, Tennessee, United States

Site Status

Houston Methodist Research Institute

Houston, Texas, United States

Site Status

University of Texas, Health Science Center

Houston, Texas, United States

Site Status

University of Virgina School of Medicine

Charlottesville, Virginia, United States

Site Status

Children's Hospital of The King's Daughter

Norfolk, Virginia, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

American Family Children's Hospital

Madison, Wisconsin, United States

Site Status

Hospital General de Niños Pedro de Elizalde

Buenos Aires, Buenos Aires F.D., Argentina

Site Status

Hospital de Niños Dr. Ricardo Gutierrez

Buenos Aires, , Argentina

Site Status

Hospital Privado Universitario de Córdoba

Córdoba, , Argentina

Site Status

Hospital Universitário Alcides Carneiro (HUAC)

São José, Campina Grande - PB, Brazil

Site Status

Instituto de Medicina Avancada (IMA Brasil)

São Paulo, São Paulo, Brazil

Site Status

CETI - Centro de Estudos em Terapias Inovadoras

Curitiba, , Brazil

Site Status

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

São Paulo, , Brazil

Site Status

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

Site Status

Bone Research and Education Centre

Oakville, Ontario, Canada

Site Status

Shiner's Hospital for Children - Canada

Montreal, Quebec, Canada

Site Status

Children's Hospital Eastern Ontario Research Institute

Ottawa, , Canada

Site Status

Pontificia Universidad Católica de Chile

Santiago, Santiago Metropolitan, Chile

Site Status

Hospital Universitario de San Vicente Fundación

Medellín, Antioquia, Colombia

Site Status

Hospital Infantil Universitario de San José

Bogotá, Cundinamarca, Colombia

Site Status

Countries

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United States Argentina Brazil Canada Chile Colombia

References

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Carpenter TO, Fukumoto S, Haffner D, Imel EA, Ozono K, Ishii H, Li Z, Sandilands K, Joos-Vandewalle P, Lee C, Kanematsu M, McCullough KP, Brandi ML. Advancing Patient Evidence in XLH (APEX): Baseline analysis of a global data unification program. Bone. 2025 Dec;201:117649. doi: 10.1016/j.bone.2025.117649. Epub 2025 Sep 15.

Reference Type DERIVED
PMID: 40962182 (View on PubMed)

Related Links

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Other Identifiers

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UX023-CL401

Identifier Type: -

Identifier Source: org_study_id

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