Clinical trial NCT04783428: Tumor-induced Osteomalacia Disease Monitoring Program
Official title: Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Tumor-induced Osteomalacia (TIO)
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2021-03-05
Enrollment: 22
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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 maybe untreated, at any time during the TIO DMP.

Brief 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Serum 1,25(OH)2D Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Alkaline Phosphatase (ALP) Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Serum FGF23 Over Time in Participants Not Undergoing Treatment With Burosumab
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Phosphaturic Mesenchymal Tumor (PMT) Size Over Time as Assessed by Tumor Imaging
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Number of Participants With New PMT Development as Assessed by Tumor Imaging
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Serum iPTH Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Urine Calcium Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time
timeFrame: 10 years
measure: Long-Term Safety of Burosumab: Number of Participants With Normal and/or Potentially Clinically Significant Pregnancy Outcomes
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Scores in Pediatric Participants Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change From Baseline in Short Form-36 version 2 (SF-36v2) in Adult Participants Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings
timeFrame: 10 years
measure: Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Bloomington
country: United States
facility: Indiana University
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Buenos Aires
country: Argentina
facility: IDIM - Instituto de Diagnóstico e Investigaciones Metabólicas
Study officials:
affiliation: Ultragenyx Pharmaceutical Inc
name: Medical Director
role: STUDY_DIRECTOR
References: