Status:
COMPLETED
Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
Lead Sponsor:
University of South Florida
Collaborating Sponsors:
National Institutes of Health (NIH)
Conditions:
Urea Cycle Disorders
Eligibility:
All Genders
Brief Summary
The purpose of this study is to determine if patients participating in "The Longitudinal Study of Urea Cycle Disorders" are different than participants in the Urea Cycles Disorders Consortium (UCDC) R...
Detailed Description
Participants enrolled in the RDCRN UCDC Contact Registry will receive an email inviting them to participate in the study. Interested participants will be directed to the informed consent document. Aft...
Eligibility Criteria
Inclusion
- Enrollment in UCDC RDCRN Contact Registry
- Patient reported diagnosis of one of the eight UCDs or UCD diagnosis highly likely/pending.
Exclusion
- Cases of hyperammonemia caused by an organic acidemia, lysinuric protein intolerance, mitochondrial disorders, congenital lactic acidemia, fatty acid oxidation defects and primary liver disease.
- Individuals with rare and unrelated serious comorbidities, e.g., Down syndrome, intraventricular hemorrhage in the newborn period, and extreme low birth weight (\<1,500 grams).
- Inability to provide informed consent and complete surveys
Key Trial Info
Start Date :
October 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
July 1 2016
Estimated Enrollment :
87 Patients enrolled
Trial Details
Trial ID
NCT02311283
Start Date
October 1 2014
End Date
July 1 2016
Last Update
March 7 2018
Active Locations (1)
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1
University of South Florida
Tampa, Florida, United States, 33612