Tumor-induced Osteomalacia Disease Monitoring Program

N/AActive Not RecruitingNCT04783428

Ultragenyx Pharmaceutical Inc23 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Tumor-induced Osteomalacia (TIO)

Interventions

No interventionOTHER

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

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (6)

Yale University

New Haven, Connecticut, United States

Indiana University

Bloomington, Indiana, United States

Johns Hopkins University

Baltimore, Maryland, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Outcomes

Primary Outcomes

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

Time frame: 10 years

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

Time frame: 10 years

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

Time frame: 10 years

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

Time frame: 10 years

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

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

Time frame: 10 years

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

Time frame: 10 years

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

Time frame: 10 years

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

Time frame: 10 years

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

Time frame: 10 years

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

Data from ClinicalTrials.gov

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