Who is eligible for clinical trial NCT04783428?

Participants must meet protocol criteria for Tumor-induced Osteomalacia (TIO). Confirm exact inclusion and exclusion requirements with the study coordinator.

Does clinical trial NCT04783428 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2026-06-02

Tumor-induced Osteomalacia Disease Monitoring Program (NCT04783428)

🔵 Active, Not Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Ultragenyx Pharmaceutical Inc

🩺 Plain-English Summary (8th-Grade Level)

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.

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (5 items) and avoid the listed exclusions (6 items).

Location: Yale University — New Haven, Connecticut

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

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

Usually NOT eligible if any of the following apply:

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

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (6 Sites)

Yale University — New Haven, Connecticut
Indiana University — Bloomington, Indiana
Johns Hopkins University — Baltimore, Maryland
Vanderbilt University Medical Center — Nashville, Tennessee
University of Virginia — Charlottesville, Virginia
IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas — Buenos Aires,

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

🏛️ View Official NIH Record ↗