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NCT04783428Active, not recruiting

Tumor-induced Osteomalacia Disease Monitoring Program

Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)

Source: ClinicalTrials.gov · Last updated 4 Nov 2026

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Study type
Observational
Enrollment
23 (actual)
First submitted
18 Feb 2021
Start date
31 Jan 2022
Primary completion date
28 Feb 2032
Lead sponsor
Ultragenyx Pharmaceutical Inc
NCT number
NCT04783428

What you commit to

  • Study type: Observational
  • Participants enrolled: 23
  • From 31 Jan 2022 to the primary completion date, 28 Feb 2032
  • 6 sites in 2 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Concurrent enrollment in a clinical trial without prior approval from the TIO DMP Sponsor
  • Undergoing treatment with burosumab for an unapproved indication

Other illness

  • 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

Other

  • Have a clinical diagnosis of TIO deemed to be caused by a tumor other than a PMT
  • Less than 1 year of life expectancy (for any cause) in the opinion of the Investigator

Who the study is looking for

  • 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

Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (6)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas
Coordinator of this site? Confirm its status
Buenos AiresArgentinaUnknown status
Johns Hopkins University
Coordinator of this site? Confirm its status
BaltimoreUnited StatesUnknown status
Indiana University
Coordinator of this site? Confirm its status
BloomingtonUnited StatesUnknown status
University of Virginia
Coordinator of this site? Confirm its status
CharlottesvilleUnited StatesUnknown status
Vanderbilt University Medical Center
Coordinator of this site? Confirm its status
NashvilleUnited StatesUnknown status
Yale University
Coordinator of this site? Confirm its status
New HavenUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.