Tumor-induced Osteomalacia Disease Monitoring Program

NCT04783428 | Active Not Recruiting FDA Drug

• 1 other identifier: UX023T-CL403
• Study Type: observational
• Target: 23
• Locations: 2 countries
• Sites: 6

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.

Conditions

Tumor-induced Osteomalacia (TIO)

Outcome Measures

Primary Outcomes (26)

• 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

  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

Study Arms (3)

Prior TIO Burosumab Clinical Trial Participants

Other: No intervention

Adults Who Have Not Participated In Prior Burosumab Clinical Trials

Other: No intervention

Pediatrics Who Have Not Participated In Prior Burosumab Clinical Trials

Other: No intervention

Interventions

No intervention OTHER

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

Adults Who Have Not Participated In Prior Burosumab Clinical Trials; Pediatrics Who Have Not Participated In Prior Burosumab Clinical Trials; Prior TIO Burosumab Clinical Trial Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ultragenyx Pharmaceutical Inc: lead

Study Sites (6)

Yale University

New Haven, Connecticut, 06520, United States

Location

Indiana University

Bloomington, Indiana, 47405, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21218, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37235, United States

Location

University of Virginia

Charlottesville, Virginia, 22908, United States

Location

IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas

Buenos Aires, Argentina

Location

Related Links

• Ultragenyx Patient Advocacy/TIO Disease Information
• Ultragenyx Transparency Commitment

MeSH Terms

Conditions

Oncogenic osteomalacia

Study Officials

• Medical Director

  Ultragenyx Pharmaceutical Inc

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2021
• First Posted: March 5, 2021
• Study Start: January 31, 2022
• Primary Completion (Estimated): February 28, 2032
• Study Completion (Estimated): February 28, 2032
• Last Updated: January 21, 2026

Record last verified: 2026-01

Locations

US (5); AR (1)