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Tumor-induced Osteomalacia Disease Monitoring Program

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04783428
Recruitment Status : Recruiting
First Posted : March 5, 2021
Last Update Posted : March 4, 2022
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Study Description
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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.

Condition or disease Intervention/treatment
Tumor-induced Osteomalacia (TIO) Other: No intervention

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
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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Tumor-induced Osteomalacia Disease Monitoring Program (TIO DMP)
Actual Study Start Date : January 31, 2022
Estimated Primary Completion Date : February 28, 2032
Estimated Study Completion Date : February 28, 2032

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Prior TIO Burosumab Clinical Trial Participants Other: No intervention
Access to any treatment is through authorized commercial use and not as part of this DMP
Adults Who Have Not Participated In Prior Burosumab Clinical Trials Other: No intervention
Access to any treatment is through authorized commercial use and not as part of this DMP
Pediatrics Who Have Not Participated In Prior Burosumab Clinical Trials Other: No intervention
Access to any treatment is through authorized commercial use and not as part of this DMP


Outcome Measures
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Primary Outcome Measures :
  1. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Phosporus Over Time [ Time Frame: 10 years ]
  2. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum 1,25(OH)2D Over Time [ Time Frame: 10 years ]
  3. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum Alkaline Phosphatase (ALP) Over Time [ Time Frame: 10 years ]
  4. Long-Term Effectiveness of Burosumab: Change From Baseline in Serum FGF23 Over Time in Participants Not Undergoing Treatment With Burosumab [ Time Frame: 10 years ]
  5. 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 ]
  6. Long-Term Safety of Burosumab: Number of Participants With New PMT Development as Assessed by Tumor Imaging [ Time Frame: 10 years ]
  7. Long-Term Safety of Burosumab: Change From Baseline in Serum iPTH Over Time [ Time Frame: 10 years ]
  8. Long-Term Safety of Burosumab: Change From Baseline in Serum Calcium Over Time [ Time Frame: 10 years ]
  9. Long-Term Safety of Burosumab: Change From Baseline in Urine Calcium Over Time [ Time Frame: 10 years ]
  10. Long-Term Safety of Burosumab: Change From Baseline Urinary Calcium/Creatinine Ratio [ Time Frame: 10 years ]
  11. Long-Term Safety of Burosumab: Change From Baseline in Serum Creatinine Over Time [ Time Frame: 10 years ]
  12. Long-Term Safety of Burosumab: Change From Baseline in Urine Creatinine Over Time [ Time Frame: 10 years ]
  13. Long-Term Safety of Burosumab: Change From Baseline in Urine Protein/Creatinine Ratio Over Time [ Time Frame: 10 years ]
  14. Long-Term Safety of Burosumab: Number of Participants With Nephrocalcinosis Over Time [ Time Frame: 10 years ]
  15. Long-Term Safety of Burosumab: Number of Participants With Serious Adverse Events (SAEs) and Adverse Events (AEs) and Related AEs [ Time Frame: 10 years ]
  16. Long-Term Safety of Burosumab: Number of Participants With Incidence and/or Progression of Spinal Stenosis Over Time [ Time Frame: 10 years ]
  17. 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

  18. Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Fatigue Inventory (BFI) Scores in Adult Participants Over Time [ Time Frame: 10 years ]
  19. 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 ]
  20. Long-Term Effectiveness of Burosumab: Change From Baseline in Brief Pain Inventory (BPI) Scores in Adult Participants Over Time [ Time Frame: 10 years ]
  21. Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Pain Scores in Pediatric Participants Over Time [ Time Frame: 10 years ]
  22. Long-Term Effectiveness of Burosumab: Change From Baseline in PROMIS Physical Function Scores Over Time [ Time Frame: 10 years ]
  23. 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 ]
  24. Long-Term Effectiveness of Burosumab: Change in Short Form-10 (SF-10) for Pediatric Participants Over Time [ Time Frame: 10 years ]
  25. Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Clinical Findings [ Time Frame: 10 years ]
  26. Long-Term Effectiveness of Burosumab: Number of Participants With Changes From Baseline in Resource/Health Utilization [ Time Frame: 10 years ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04783428


Contacts
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Contact: Patients Contact: Trial Recruitment 1-888-756-8657 trialrecruitment@ultragenyx.com
Contact: HCPs Contact: Medical Information 1-888-756-8657 medinfo@ultragenyx.com

Locations
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United States, Connecticut
Yale University Not yet recruiting
New Haven, Connecticut, United States, 06520
United States, Indiana
Indiana University Not yet recruiting
Bloomington, Indiana, United States, 47405
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21218
United States, Tennessee
Vanderbilt University Medical Center Not yet recruiting
Nashville, Tennessee, United States, 37235
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc
More Information
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Additional Information:

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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT04783428    
Other Study ID Numbers: UX023T-CL403
First Posted: March 5, 2021    Key Record Dates
Last Update Posted: March 4, 2022
Last Verified: March 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Neoplasms, Connective Tissue
Osteomalacia
Rickets
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Metabolic Diseases
Calcium Metabolism Disorders
Vitamin D Deficiency
 Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Connective Tissue Diseases