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X-linked Hypophosphatemia Disease Monitoring Program

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ClinicalTrials.gov Identifier: NCT03651505
Recruitment Status : Recruiting
First Posted : August 29, 2018
Last Update Posted : October 14, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

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.

Condition or disease Intervention/treatment
X-linked Hypophosphatemia Hypophosphatemic Rickets Other: No intervention

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.

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Study Type : Observational
Estimated Enrollment : 500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
Actual Study Start Date : July 16, 2018
Estimated Primary Completion Date : July 2028
Estimated Study Completion Date : July 2028


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




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Approximately 500 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.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03651505


Contacts
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Contact: Patients Contact: Patient Advocacy 1-415-483-8800 patientadvocacy@ultragenyx.com
Contact: HCPs Contact: Medical Information 1-888-756-8657 medinfo@ultragenyx.com

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Locations
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United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Rady Children's Hospital San Diego Recruiting
San Diego, California, United States, 92123
University of California San Francisco Recruiting
San Francisco, California, United States, 94158
United States, Colorado
Children's Hospital of Colorado Recruiting
Aurora, Colorado, United States, 80045
United States, Connecticut
Yale University School of Medicine Recruiting
New Haven, Connecticut, United States, 06520
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
United States, Indiana
Indiana University School of Medicine Recruiting
Indianapolis, Indiana, United States, 46202-5167
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21218
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27708
United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
The Ohio State University Wexner Medical Center Recruiting
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Vanderbilt University - Department of Medicine Recruiting
Nashville, Tennessee, United States, 37212
Vanderbilt University - Department of Pediatrics Recruiting
Nashville, Tennessee, United States, 37232
United States, Texas
Houston Methodist Research Institute Recruiting
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
United States, Wisconsin
American Family Children's Hospital Recruiting
Madison, Wisconsin, United States, 53792
Argentina
Hospital de Niños Dr. Ricardo Gutierrez Not yet recruiting
Buenos Aires, Argentina, C1425EFD
Brazil
Serviço de Endocrinologia e Metabologia - Hospital de Clínicas da Universidade Federal do Para (SEMPR) Recruiting
Curitiba, Parana, Brazil, 80030-110
Instituto de Medicina Avancada (IMA Brasil) Recruiting
São Paulo, Sao Paulo, Brazil, 05437-000
Canada
Children's Hospital Eastern Ontario Research Institute Recruiting
Ottawa, Canada, K1H 5B2
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Ultragenyx Medical Director Ultragenyx Pharmaceuticals Inc.

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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03651505     History of Changes
Other Study ID Numbers: UX023-CL401
First Posted: August 29, 2018    Key Record Dates
Last Update Posted: October 14, 2019
Last Verified: October 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ultragenyx Pharmaceutical Inc:
X-linked Hypophosphatemia
Fibroblast growth factor 23 (FGF23)
KRN23
XLH
Hypophosphatemia
Familial Hypophosphatemic Rickets
FGF23
Crysvita
Additional relevant MeSH terms:
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Rickets
Familial Hypophosphatemic Rickets
Rickets, Hypophosphatemic
Hypophosphatemia
Phosphorus Metabolism Disorders
Metabolic Diseases
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Calcium Metabolism Disorders
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs