X-linked Hypophosphatemia Disease Monitoring Program



Status:Recruiting
Conditions:Gastrointestinal, Metabolic
Therapuetic Areas:Gastroenterology, Pharmacology / Toxicology
Healthy:No
Age Range:Any
Updated:3/21/2019
Start Date:July 16, 2018
End Date:October 2028
Contact:Patients Contact: Patient Advocacy
Email:patientadvocacy@ultragenyx.com
Phone:1-415-483-8800

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X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)

The objectives of this observational study are to characterize XLH disease presentation and
progression and to assess long-term effectiveness and safety of burosumab.

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.

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 five years of life expectancy.
We found this trial at
17
sites
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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3400 N Charles St
Baltimore, Maryland 21205
410-516-8000
Johns Hopkins University The Johns Hopkins University opened in 1876, with the inauguration of its...
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Baltimore, MD
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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Durham, North Carolina 27710
(919) 684-8111
Duke University Younger than most other prestigious U.S. research universities, Duke University consistently ranks among...
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Durham, NC
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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3020 Childrens way
San Diego, California 92123
(858) 576-1700
Rady Children's Hospital - San Diego Rady Children's Hospital-San Diego is the region’s pediatric medical...
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San Diego, CA
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Aurora, CO
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Chicago, IL
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Cincinnati, Ohio 45229
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Cincinnati, OH
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410 W 10th Ave
Columbus, Ohio 43210
(614) 293-8652
The Ohio State University, Wexner Medical Center Located in Columbus, The Ohio State University Wexner...
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Columbus, OH
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Houston, TX
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340 W 10th St #6200
Indianapolis, Indiana 46202
(317) 274-3772
Indiana University School of Medicine With more than 2,000 students in 2013, the Indiana University...
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Indianapolis, IN
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Montréal,
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Nashville, TN
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333 Cedar St
New Haven, Connecticut 06504
(203) 432-4771
Yale University School of Medicine Founded in 1810, the Yale School of Medicine is a...
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New Haven, CT
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San Francisco, California 94143
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San Francisco, CA
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Seattle, WA
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