Tumor-induced Osteomalacia Disease Monitoring Program | Clinical Trial | StuddyBuddy@endsection
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Active Not Recruiting
NCT04783428
Tumor-induced Osteomalacia Disease Monitoring Program
Conditions: Tumor-induced Osteomalacia (TIO)
Sex: All
Healthy volunteers: No
Enrollment: 23
Sponsor: Ultragenyx Pharmaceutical Inc
Location: Yale University New Haven Connecticut
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 physician and to monitor the course of the underlying phosphaturic mesenchymal tumor (PMT) overtime in patients with TIO irrespective of their treatment status.
Eligibility Criteria
Inclusion Criteria:
* 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 Criteria:
* 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
Source: ClinicalTrials.gov (NCT04783428). StuddyBuddy aggregates publicly available trial information.