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X-linked Hypophosphatemia Disease Monitoring Program

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ClinicalTrials.gov Identifier: NCT03651505
Recruitment Status : Recruiting
First Posted : August 29, 2018
Last Update Posted : October 14, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Tracking Information
First Submitted Date August 24, 2018
First Posted Date August 29, 2018
Last Update Posted Date October 14, 2019
Actual Study Start Date July 16, 2018
Estimated Primary Completion Date July 2028   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 28, 2018)
  • Long-Term Safety of Burosumab [ Time Frame: 10 years ]
    To assess the long-term safety of burosumab treatment in adult and pediatric patients with XLH, including overall renal health, the presence and/or progression of nephrocalcinosis and spinal stenosis, and pregnancy outcomes.
  • Long-Term Effectiveness of Burosumab [ Time Frame: 10 years ]
    To evaluate the long-term effectiveness of burosumab treatment on key manifestations of XLH, including skeletal health, stiffness, mobility and physical functioning.
  • Clinical Course of XLH Disease [ Time Frame: 10 years ]
    To illustrate the clinical, radiological, biochemical manifestations and progression of XLH over time in both untreated and treated patients with XLH.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03651505 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title X-linked Hypophosphatemia Disease Monitoring Program
Official Title X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
Brief Summary The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
Detailed Description The XLH-DMP is a global, prospective, multicenter, longitudinal, long-term outcomes program for subjects on or off any treatment designed to characterize XLH disease presentation and progression, assess long-term safety and effectiveness of burosumab, as well as prospectively investigate longitudinal change over time across biomarker(s), clinical assessments, and patient/caregiver-reported outcome measures in a representative population. The XLH-DMP will collect demographic, biochemical, physiologic, disease severity, and progression data in patients taking burosumab and those not taking burosumab. In this DMP, patients will only have access to burosumab through authorized prescribed use. The Sponsor will not provide any treatments as part of the DMP.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Approximately 500 eligible adult and pediatric patients with XLH will be enrolled globally, with a minimum of 200 pediatric patients. Patients can enter the XLH-DMP regardless of how their XLH is being treated. Patients on Crysvita (burosumab) via prescription may begin taking Crysvita, per standard of care before or after enrolling in the XLH DMP.
Condition
  • X-linked Hypophosphatemia
  • Hypophosphatemic Rickets
Intervention Other: No intervention
Access to any treatment is through authorized commercial use and not as a part of this DMP
Study Groups/Cohorts
  • Prior Burosumab Clinical Trial Participants
    Patients who participated in burosumab clinical trials and continue to receive burosumab via prescription from their physician.
    Intervention: Other: No intervention
  • Not from Prior Burosumab Clinical Trial
    Patients may take other treatments for XLH and may start burosumab treatment at any time as prescribed by a physician.
    Intervention: Other: No intervention
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: August 28, 2018)
500
Original Estimated Enrollment Same as current
Estimated Study Completion Date July 2028
Estimated Primary Completion Date July 2028   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Willing and able to provide informed consent or, in the case of patients under the age of 18 years (or 16 years, depending on the region), provide assent (if required) and informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
  • Clinical diagnosis of XLH based on family history, OR confirmed PHEX mutation, OR biochemical profile consistent with XLH.
  • Willing and able to comply with the study visit schedule and study procedures.

Exclusion Criteria:

  • Concurrent enrollment in an Ultragenyx-sponsored clinical trial is NOT permitted.
  • Serious medical or psychiatric comorbidity.
  • Less than one year of life expectancy.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Patients Contact: Patient Advocacy 1-415-483-8800 patientadvocacy@ultragenyx.com
Contact: HCPs Contact: Medical Information 1-888-756-8657 medinfo@ultragenyx.com
Listed Location Countries Argentina,   Brazil,   Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03651505
Other Study ID Numbers UX023-CL401
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Ultragenyx Pharmaceutical Inc
Study Sponsor Ultragenyx Pharmaceutical Inc
Collaborators Not Provided
Investigators
Study Director: Ultragenyx Medical Director Ultragenyx Pharmaceuticals Inc.
PRS Account Ultragenyx Pharmaceutical Inc
Verification Date October 2019