Longitudinal Study of Urea Cycle Disorders
NCT00237315 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 1500
Last updated 2024-02-13
Summary
Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy, and coma. The purpose of this study is to perform a long-term analysis of a large group of individuals with various UCDs. The study will focus on the natural history, disease progression, treatment, and outcome of individuals with UCD.
Conditions
- Brain Diseases, Metabolic, Inborn
- Amino Acid Metabolism, Inborn Errors
- Urea Cycle Disorders
Sponsors & Collaborators
-
National Center for Research Resources (NCRR)
collaborator NIH -
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
collaborator NIH -
Rare Diseases Clinical Research Network
collaborator NETWORK -
Andrea Gropman
lead OTHER
Principal Investigators
-
Andrea Gropman, MD · Children's National Research Institute
-
Susan Berry, MD · University of Minnesota Masonic Children's Hospital
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2006-02-28
- Primary Completion
- 2025-07-31
- Completion
- 2026-07-31
Countries
- United States
- Canada
- Germany
- Switzerland
Study Locations
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