Tumor-induced Osteomalacia Disease Monitoring Program

Tumor-induced Osteomalacia (TIO) Clinical Trial

Official title:

Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04783428
• Other study ID #: UX023T-CL403
• Status: Active, not recruiting
• Start date: January 31, 2022
• Est. completion date: February 28, 2032

Study information

• Verified date: May 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Active, not recruiting
• Enrollment: 21
• Est. completion date: February 28, 2032
• Est. primary completion date: February 28, 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Neoplasms, Connective Tissue
• Osteomalacia
• Tumor-induced Osteomalacia (TIO)

Intervention

Other:
• No intervention
Access to any treatment is through authorized commercial use and not as part of this DMP

Locations

Country	Name	City	State
Argentina	IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas	Buenos Aires
United States	Johns Hopkins University	Baltimore	Maryland
United States	Indiana University	Bloomington	Indiana
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Yale University	New Haven	Connecticut

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Argentina, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Serum 1,25(OH)2D Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Alkaline Phosphatase (ALP) Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Serum FGF23 Over Time in Participants Not Undergoing Treatment With Burosumab		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Phosphaturic Mesenchymal Tumor (PMT) Size Over Time as Assessed by Tumor Imaging		10 years
Primary	Long-Term Safety of Burosumab: Number of Participants With New PMT Development as Assessed by Tumor Imaging		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Serum iPTH Over Time		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium Over Time		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Urine Calcium Over Time		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time		10 years
Primary	Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time		10 years
Primary	Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time		10 years
Primary	Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs		10 years
Primary	Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time		10 years
Primary	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
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time		10 years
Primary	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
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change From Baseline in Short Form-36 version 2 (SF-36v2) in Adult Participants Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time		10 years
Primary	Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings		10 years
Primary	Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization		10 years

External Links

•   Ultragenyx Patient Advocacy/TIO Disease Information