Longitudinal Study of Urea Cycle Disorders
Part of paid clinical trials in Los Angeles, California.
- Sponsor
- Andrea Gropman
- Study ID
- NCT00237315
- Status
- Recruiting
Conditions
- Amino Acid Metabolism, Inborn Errors
- Brain Diseases, Metabolic, Inborn
- Urea Cycle Disorders
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Study Details
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.
Key Dates
- Start date
- Feb 28, 2006
- Status verified
- Feb 2024
- Primary completion
- Jul 31, 2025
- Completion
- Jul 31, 2026
Study Design
- Enrollment
- 1,500 participants (estimated)
Primary Outcome Measure
Prevalence of specific morbid indicators of disease severity [ Time Frame: End of study ]
Central Contacts
- Jennifer Seminara, MPH202-306-6489
Locations (12)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | 90095 | Stephen Cederbaum, MD (SUB_INVESTIGATOR) Derek Wong, MD (PRINCIPAL_INVESTIGATOR) |
| Stanford University Medical Center | Stanford | California | 94305 | Gregory Enns, MD (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | Shawn McCandless, MD (PRINCIPAL_INVESTIGATOR) Curtis Coughlin, MS, CGC (PRINCIPAL_INVESTIGATOR) Johan Van Hove, MD, PhD (SUB_INVESTIGATOR) Greta Wilkening, PhD (SUB_INVESTIGATOR) Janet Thomas, MD (SUB_INVESTIGATOR) |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | Nicholas Ah Mew, MD (PRINCIPAL_INVESTIGATOR) Jacqueline Sanz, PhD (SUB_INVESTIGATOR) |
| Children's Hospital Boston (UCDC New England Center) | Boston | Massachusetts | 02115 | Gerard Berry, MD (PRINCIPAL_INVESTIGATOR) Harvey Levy, MD (SUB_INVESTIGATOR) |
| University of Minnesota | Minneapolis | Minnesota | 55455 | Susan Berry, MD (PRINCIPAL_INVESTIGATOR) |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | Margo Breilyn, MD (PRINCIPAL_INVESTIGATOR) |
| Case Western Medical College | Cleveland | Ohio | 44106 | Laura Konczal, MD (PRINCIPAL_INVESTIGATOR) Rachel Tangen, PhD (SUB_INVESTIGATOR) Lori-Anne Schillaci, MD (SUB_INVESTIGATOR) |
| Oregon Health and Science University | Portland | Oregon | 97239 | Cary Harding, MD (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Can Ficicioglu, MD (PRINCIPAL_INVESTIGATOR) |
| Baylor College of Medicine | Houston | Texas | 77030 | Sandesh Nagamani, MD (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital and Regional Medical Center | Seattle | Washington | 98105 | Christina Lam, MD, PhD (PRINCIPAL_INVESTIGATOR) |
Find similar trials in Los Angeles, CA
By research site
University of California, Los Angeles· Los Angeles, CAStanford University Medical Center· Stanford, CAChildren's Hospital Colorado· Aurora, COChildren's National Medical Center· Washington D.C., DCChildren's Hospital Boston (UCDC New England Center)· Boston, MAUniversity of Minnesota· Minneapolis, MN
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