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X-linked Hypophosphatemia Disease Monitoring Program

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03651505
Recruitment Status : Active, not recruiting
First Posted : August 29, 2018
Last Update Posted : March 22, 2023
Kyowa Kirin Co., Ltd.
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
Actual Enrollment : 780 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 : December 2032
Estimated Study Completion Date : December 2032

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.

Information from the National Library of Medicine

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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 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.

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

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Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Kyowa Kirin Co., Ltd.
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Study Director: Ultragenyx Medical Director Ultragenyx Pharmaceuticals Inc.
Additional Information:
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03651505    
Other Study ID Numbers: UX023-CL401
First Posted: August 29, 2018    Key Record Dates
Last Update Posted: March 22, 2023
Last Verified: March 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Ultragenyx Pharmaceutical Inc:
X-linked Hypophosphatemia
Fibroblast growth factor 23 (FGF23)
Familial Hypophosphatemic Rickets
Additional relevant MeSH terms:
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Familial Hypophosphatemic Rickets
Rickets, Hypophosphatemic
Phosphorus Metabolism Disorders
Metabolic Diseases
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Calcium Metabolism Disorders
Vitamin D Deficiency
Deficiency Diseases
Nutrition Disorders
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn