Status:
ACTIVE_NOT_RECRUITING
Tumor-induced Osteomalacia Disease Monitoring Program
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Tumor-induced Osteomalacia (TIO)
Eligibility:
All Genders
Brief Summary
The objectives of this observational study are to assess the long-term safety and long-term effectiveness of burosumab in patients with TIO who are being treated with burosumab as prescribed by their ...
Detailed Description
Enrolled patients may or may not be treated with commercially available burosumab during the TIO DMP at the discretion of their treating physician. Given the observational nature of the TIO DMP, speci...
Eligibility Criteria
Inclusion
- Have a clinical diagnosis of TIO based on the presence of an underlying PMT (confirmed by imaging) AND/OR historical documentation. Note: For adult patients with TIO in whom the causative PMT has never been located, and all pediatric patients, documented evidence of negative genetic testing for other hereditary hypophosphatemic disorders is necessary
- For patient safety, all participating female patients of child-bearing potential must be willing to have pregnancy tests prior to certain assessments performed as part of the DMP
- Be willing to provide access to prior medical records including tumor pathology reports and biopsy slides, imaging, biochemical, and diagnostic, medical, and surgical history data, if available
- Be willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related procedures
- Be willing and able to comply with the study visit schedule and study procedures
Exclusion
- Have a clinical diagnosis of TIO deemed to be caused by a tumor other than a PMT
- Serious medical or psychiatric comorbidity that, in the opinion of the Investigator, would present a concern for patient safety or compromise the ability to provide consent or comply with the study visit schedule and study procedures
- Less than 1 year of life expectancy (for any cause) in the opinion of the Investigator
- Concurrent enrollment in a clinical trial without prior approval from the TIO DMP Sponsor
- Undergoing treatment with burosumab for an unapproved indication
Key Trial Info
Start Date :
January 31 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
February 28 2032
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT04783428
Start Date
January 31 2022
End Date
February 28 2032
Last Update
September 19 2025
Active Locations (6)
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1
Yale University
New Haven, Connecticut, United States, 06520
2
Indiana University
Bloomington, Indiana, United States, 47405
3
Johns Hopkins University
Baltimore, Maryland, United States, 21218
4
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37235