Tumor-induced Osteomalacia Disease Monitoring Program
Active, not recruiting
- Conditions
- Tumor-induced Osteomalacia (TIO)
- Interventions
- Other: No intervention
- Registration Number
- NCT04783428
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- 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 physician and to monitor the course of the underlying phosphaturic mesenchymal tumor (PMT) overtime in patients with TIO irrespective of their treatment status.
- 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, specific treatments or supportive management will not be provided as part of the study.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 21
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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Prior TIO Burosumab Clinical Trial Participants No intervention - Adults Who Have Not Participated In Prior Burosumab Clinical Trials No intervention - Pediatrics Who Have Not Participated In Prior Burosumab Clinical Trials No intervention -
- Primary Outcome Measures
Name Time Method Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants 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 in Urine Creatinine Over Time 10 years Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time 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 Urine Protein/Creatinine Ratio Over Time 10 years Long-Term Safety of Burosumab: Number of Participants With Normal and/or Potentially Clinically Significant Pregnancy Outcomes 10 years Includes maternal, neonatal and infant outcomes
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 Serum iPTH Over Time 10 years Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio 10 years Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time 10 years Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time 10 years Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time 10 years Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium 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 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: Number of Participants With Changes From Baseline in Resource/Health Utilization 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
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (5)
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Indiana University
🇺🇸Bloomington, Indiana, United States
Yale University
🇺🇸New Haven, Connecticut, United States
IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas
🇦🇷Buenos Aires, Argentina