X-linked Hypophosphatemia Disease Monitoring Program
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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 |
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Recruitment Status :
Active, not recruiting
First Posted : August 29, 2018
Last Update Posted : March 22, 2023
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Sponsor:
Ultragenyx Pharmaceutical Inc
Collaborator:
Kyowa Kirin Co., Ltd.
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc
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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 |
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.
| 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 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hereditary hypophosphatemic rickets
| Group/Cohort | Intervention/treatment |
|---|---|
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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.
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Other: No intervention
Access to any treatment is through authorized commercial use and not as a part of this DMP |
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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.
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Other: No intervention
Access to any treatment is through authorized commercial use and not as a part of this DMP |
Primary Outcome Measures :
- 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.
- 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.
- 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 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.
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
Locations
Show 39 study locations
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Kyowa Kirin Co., Ltd.
Investigators
| Study Director: | Ultragenyx Medical Director | Ultragenyx Pharmaceuticals Inc. |
Additional Information:
| 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 |
| 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:
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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 |