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Active, Not Recruiting

NCT Number: NCT04783428

Tumor-induced Osteomalacia Disease Monitoring Program

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Tumor-induced Osteomalacia (TIO) Oncogenic osteomalacia

Sex eligibility

All sexes

Study type

Observational

Primary location

IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas, Buenos Aires, Argentina

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

No intervention

Other

Access to any treatment is through authorized commercial use and not as part of this DMP

Primary outcomes

  1. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time

    Time frame: 10 years

  2. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum 1,25(OH)2D Over Time

    Time frame: 10 years

  3. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Alkaline Phosphatase (ALP) Over Time

    Time frame: 10 years

  4. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum FGF23 Over Time in Participants Not Undergoing Treatment With Burosumab

    Time frame: 10 years

  5. Long-Term Safety of Burosumab: Change From Baseline in Phosphaturic Mesenchymal Tumor (PMT) Size Over Time as Assessed by Tumor Imaging

    Time frame: 10 years

  6. Long-Term Safety of Burosumab: Number of Participants With New PMT Development as Assessed by Tumor Imaging

    Time frame: 10 years

  7. Long-Term Safety of Burosumab: Change From Baseline in Serum iPTH Over Time

    Time frame: 10 years

  8. Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium Over Time

    Time frame: 10 years

  9. Long-Term Safety of Burosumab: Change From Baseline in Urine Calcium Over Time

    Time frame: 10 years

  10. Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio

    Time frame: 10 years

  11. Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time

    Time frame: 10 years

  12. Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time

    Time frame: 10 years

  13. Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time

    Time frame: 10 years

  14. Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time

    Time frame: 10 years

  15. Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs

    Time frame: 10 years

  16. Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time

    Time frame: 10 years

  17. Long-Term Safety of Burosumab: Number of Participants With Normal and/or Potentially Clinically Significant Pregnancy Outcomes

    Time frame: 10 years

    Includes maternal, neonatal and infant outcomes

  18. Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time

    Time frame: 10 years

  19. Long-Term Effectiveness of Burosumab: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Scores in Pediatric Participants Over Time

    Time frame: 10 years

  20. Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time

    Time frame: 10 years

  21. Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time

    Time frame: 10 years

  22. Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time

    Time frame: 10 years

  23. Long-Term Effectiveness of Burosumab: Change From Baseline in Short Form-36 version 2 (SF-36v2) in Adult Participants Over Time

    Time frame: 10 years

  24. Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time

    Time frame: 10 years

  25. Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings

    Time frame: 10 years

  26. Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization

    Time frame: 10 years

Sponsors and collaborators

Lead sponsor

Ultragenyx Pharmaceutical Inc

Industry

Registry information

Official study title

Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)

Important dates

Study start
2022
Primary completion
2032
Study completion
2032
First posted
Mar 5, 2021
Registry last updated
Jun 3, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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