Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

Completed

Conditions studied: Urea Cycle Disorders

In brief

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) Rare Diseases Clinical Research Network (RDCRN) Contact Registry and to determine if patients are a good source of medical information.

Key facts

Study ID

NCT02311283

Run by

University of South Florida

People needed

87

Starts

2014-10-01

Expected to finish

2016-07-01

Last updated by the study team

2018-03-07

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Enrollment in UCDC RDCRN Contact Registry
• Patient reported diagnosis of one of the eight UCDs or UCD diagnosis highly likely/pending.

You may not qualify if…

• 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

Where it is running

• University of South Florida — Tampa, Florida, United States

Full record on ClinicalTrials.gov

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