Tumor-induced Osteomalacia (TIO)NCT04783428
Tumor-induced Osteomalacia Disease Monitoring Program
An observational study of Tumor-induced Osteomalacia (TIO), run by Ultragenyx Pharmaceutical Inc at 6 sites in 2 countries.
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Tumor-induced Osteomalacia (TIO)
- How many people
- 23 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 31 Jan 2022to 28 Feb 2032
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
Johns Hopkins University
Baltimore, Maryland, 21218
Indiana University
Bloomington, Indiana, 47405
University of Virginia
Charlottesville, Virginia, 22908
Vanderbilt University Medical Center
Nashville, Tennessee, 37235
Yale University
New Haven, Connecticut, 06520
Argentina1 site
IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas
Buenos Aires
About this study
Official title: Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)
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.
Read the detailed 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.
- Conditions
- Tumor-induced Osteomalacia (TIO)
- Being tested
- No intervention
- Medical subject headings
- Oncogenic osteomalacia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
Access to any treatment is through authorized commercial use and not as part of this DMP
| Study group | Role | What they receive |
|---|---|---|
| Prior TIO Burosumab Clinical Trial Participants | Not described Receives: Other: No intervention | |
| Adults Who Have Not Participated In Prior Burosumab Clinical Trials | Not described Receives: Other: No intervention | |
| Pediatrics Who Have Not Participated In Prior Burosumab Clinical Trials | Not described Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Serum 1,25(OH)2D Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Alkaline Phosphatase (ALP) Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Serum FGF23 Over Time in Participants Not Undergoing Treatment With Burosumab
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Phosphaturic Mesenchymal Tumor (PMT) Size Over Time as Assessed by Tumor Imaging
Measured: 10 years
Long-Term Safety of Burosumab: Number of Participants With New PMT Development as Assessed by Tumor Imaging
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Serum iPTH Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Urine Calcium Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time
Measured: 10 years
Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs
Measured: 10 years
Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time
Measured: 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
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time
Measured: 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
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Short Form-36 version 2 (SF-36v2) in Adult Participants Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time
Measured: 10 years
Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings
Measured: 10 years
Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2021
- Study start31 Jan 2022
- Primary completion28 Feb 2032 (estimated)
- Study completion28 Feb 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tumor-induced Osteomalacia (TIO)Studies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04783428
- Sponsor's protocol ID
- UX023T-CL403
- Status last verified
- Nov 2026
- First submitted
- 18 Feb 2021
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)
- ClinicalTrials.gov ID
- NCT04783428
- Acronym / sponsor ID
- UX023T-CL403
- Conditions
- Tumor-induced Osteomalacia (TIO)
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 31 Jan 2022 (actual)
- Primary completion
- 28 Feb 2032 (estimated)
- Study completion
- 28 Feb 2032 (estimated)
- First posted
- 5 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionAccess to any treatment is through authorized commercial use and not as part of this DMP | Other | 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 |
Primary outcome
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
Time frame: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time
Time frame: 10 years
Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time
Time frame: 10 years
Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time
Time frame: 10 years
Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs
Time frame: 10 years
Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time
Time frame: 10 years
Long-Term Safety of Burosumab: Number of Participants With Normal and/or Potentially Clinically Significant Pregnancy Outcomes
Time frame: 10 years
Includes maternal, neonatal and infant outcomes
Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time
Time frame: 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
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Change From Baseline in Short Form-36 version 2 (SF-36v2) in Adult Participants Over Time
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings
Time frame: 10 years
Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization
Time frame: 10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
- Ultragenyx Patient Advocacy/TIO Disease InformationLink provided by the sponsor.
- Ultragenyx Transparency CommitmentLink provided by the sponsor.
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Tumor-Induced Osteomalacia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Ultragenyx Pharmaceutical Inc
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Tumor-induced Osteomalacia (TIO), run by Ultragenyx Pharmaceutical Inc at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Argentina. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time (time frame: 10 years). The study lists 26 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2026.