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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.
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, specific treatments or supportive management will not be provided as part of the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Prior TIO Burosumab Clinical Trial Participants |
| ||
| Adults Who Have Not Participated In Prior Burosumab Clinical Trials |
| ||
| Pediatrics Who Have Not Participated In Prior Burosumab Clinical Trials |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No intervention | Other | Access to any treatment is through authorized commercial use and not as part of this DMP |
|
| Measure | Description | Time Frame |
|---|---|---|
| Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time | 10 years | |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Serum 1,25(OH)2D Over Time | 10 years | |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Alkaline Phosphatase (ALP) Over Time | 10 years | |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Serum FGF23 Over Time in Participants Not Undergoing Treatment With Burosumab | 10 years | |
| Long-Term Safety of Burosumab: Change From Baseline in Phosphaturic Mesenchymal Tumor (PMT) Size Over Time as Assessed by Tumor Imaging | 10 years | |
| Long-Term Safety of Burosumab: Number of Participants With New PMT Development as Assessed by Tumor Imaging | 10 years | |
| Long-Term Safety of Burosumab: Change From Baseline in Serum iPTH Over Time | 10 years | |
| Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium Over Time | 10 years | |
| Long-Term Safety of Burosumab: Change From Baseline in Urine Calcium Over Time | 10 years | |
| Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio |
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Inclusion Criteria:
Exclusion Criteria:
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May include patients who have undergone complete tumor resection and continue to have biochemical/clinical evidence of disease, patients with tumor identified, or patients in whom causative tumor has not been identified and who have been diagnosed with TIO based on biochemical/clinical symptom profile.
Patients may be treated with burosumab, or phosphate and active vitamin D metabolites/analogs, as prescribed by a physician, or may be untreated, at any time during the TIO DMP.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University | New Haven | Connecticut | 06520 | United States | ||
| Indiana University |
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| Label | URL |
|---|---|
| Ultragenyx Patient Advocacy/TIO Disease Information | View source |
| Ultragenyx Transparency Commitment | View source |
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| ID | Term |
|---|---|
| C537751 | Oncogenic osteomalacia |
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| 10 years |
| Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time | 10 years |
| Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time | 10 years |
| Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time | 10 years |
| Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time | 10 years |
| Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs | 10 years |
| Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time | 10 years |
| Long-Term Safety of Burosumab: Number of Participants With Normal and/or Potentially Clinically Significant Pregnancy Outcomes | Includes maternal, neonatal and infant outcomes | 10 years |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Scores in Pediatric Participants Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Change From Baseline in Short Form-36 version 2 (SF-36v2) in Adult Participants Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time | 10 years |
| Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings | 10 years |
| Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization | 10 years |
| Bloomington |
| Indiana |
| 47405 |
| United States |
| Johns Hopkins University | Baltimore | Maryland | 21218 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37235 | United States |
| University of Virginia | Charlottesville | Virginia | 22908 | United States |
| IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas | Buenos Aires | Argentina |