X-linked Hypophosphatemia Disease Monitoring Program
Purpose
The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
Conditions
- X-linked Hypophosphatemia
- Hypophosphatemic Rickets
Eligibility
- Eligible Ages
- All ages
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Prior Burosumab Clinical Trial Participants | Patients who participated in burosumab clinical trials and continue to receive burosumab via prescription from their physician. |
|
| 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. |
|
More Details
- Status
- Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc
Study Contact
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.