Clinical trial NCT03651505: X-linked Hypophosphatemia Disease Monitoring Program
Official title: X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: X-linked Hypophosphatemia
Hypophosphatemic Rickets
Keywords: X-linked Hypophosphatemia
Fibroblast growth factor 23 (FGF23)
KRN23
XLH
Hypophosphatemia
Familial Hypophosphatemic Rickets
FGF23
Crysvita
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
Collaborators: name: Kyowa Kirin Co., Ltd.
First posted: 2018-08-29
Enrollment: 780
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief 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.

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.

Eligibility criteria: 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 outcomes:
measure: Long-Term Safety of Burosumab
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab
timeFrame: 10 years
measure: Clinical Course of XLH Disease
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's Hospital of Alabama
state: Alabama
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Karp Family Research Laboratories
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University - Department of Medicine
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University - Department of Pediatrics
state: Tennessee
city: Houston
country: United States
facility: Houston Methodist Research Institute
state: Texas
city: Houston
country: United States
facility: University of Texas, Health Science Center
state: Texas
city: Charlottesville
country: United States
facility: University of Virgina School of Medicine
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of The King's Daughter
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Madison
country: United States
facility: American Family Children's Hospital
state: Wisconsin
city: Buenos Aires
country: Argentina
facility: Hospital General de Niños Pedro de Elizalde
state: Ciudad Autónoma De Buenos Aires
city: Buenos Aires
country: Argentina
facility: Hospital de Niños Dr. Ricardo Gutierrez
city: Córdoba
country: Argentina
facility: Hospital Privado Universitario de Córdoba
city: São José
country: Brazil
facility: Hospital Universitário Alcides Carneiro (HUAC)
state: Campina Grande - PB
city: São Paulo
country: Brazil
facility: Instituto de Medicina Avancada (IMA Brasil)
state: Sao Paulo
city: Curitiba
country: Brazil
facility: CETI - Centro de Estudos em Terapias Inovadoras
city: São Paulo
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: Oakville
country: Canada
facility: Bone Research and Education Centre
state: Ontario
city: Montréal
country: Canada
facility: Shiner's Hospital for Children - Canada
state: Quebec
city: Ottawa
country: Canada
facility: Children's Hospital Eastern Ontario Research Institute
city: Santiago
country: Chile
facility: Pontificia Universidad Católica de Chile
state: Metropolitana
city: Medellín
country: Colombia
facility: Hospital Universitario de San Vicente Fundación
state: Antioquia
city: Bogotá
country: Colombia
facility: Hospital Infantil Universitario de San José
state: Cundinamarca
Study officials:
affiliation: Ultragenyx Pharmaceuticals Inc.
name: Ultragenyx Medical Director
role: STUDY_DIRECTOR
References: