X-linked Hypophosphatemia Disease Monitoring Program

X-linked Hypophosphatemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03651505
• Other study ID #: UX023-CL401
• Status: Active, not recruiting
• Start date: July 16, 2018
• Est. completion date: December 2032

Study information

• Verified date: March 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 780
• Est. completion date: December 2032
• Est. primary completion date: December 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Familial Hypophosphatemic Rickets
• Hypophosphatemia
• Hypophosphatemic Rickets
• Rickets
• X-linked Hypophosphatemia

Intervention

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

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc	Kyowa Kirin Co., Ltd.

Countries where clinical trial is conducted

United States,  Argentina,  Brazil,  Canada,  Chile,  Colombia, 

Outcome

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