Clinical trialNCT00237315UnknownObservational

Longitudinal Study of Urea Cycle Disorders

SponsorAndrea GropmanEnrollment1,500Amino Acid Metabolism, Inborn ErrorsBrain Diseases, Metabolic, InbornUrea Cycle Disorders

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Andrea Gropman, MD
Children's National Research Institute
Study Chair
clinicaltrials.gov ↗
Susan Berry, MD
University of Minnesota Masonic Children's Hospital
Study Chair
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

15 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Canada

the Hospital for Sick Children
Toronto, Ontario, Canada
Andreas Schulze, MDAnnette Feigenbaum, MD
Recruiting

Germany

University of Heidelberg
Heidelberg, Germany
Georg Hoffmann, MDPeter Burgard, PhDStefan Kölker, MD
Recruiting

Switzerland

University Children'S Hospital
Zurich, Switzerland
Matthias Baumgartner, MDTamar Stricker, MD
Recruiting

United States

University of California, Los Angeles
Los Angeles, California, United States
Derek Wong, MDStephen Cederbaum, MD
Recruiting
Stanford University Medical Center
Stanford, California, United States
Gregory Enns, MD
Recruiting
Children'S Hospital Colorado
Aurora, Colorado, United States
Curtis Coughlin, MS, CGCGreta Wilkening, PhDJanet Thomas, MDJohan Van Hove, MD, PhDShawn McCandless, MD
Recruiting
Children'S National Medical Center
Washington D.C., District of Columbia, United States
Jacqueline Sanz, PhDNicholas Ah Mew, MD
Recruiting
Children'S Hospital Boston (ucdc New England Center)
Boston, Massachusetts, United States
Gerard Berry, MDHarvey Levy, MD
Recruiting
University of Minnesota
Minneapolis, Minnesota, United States
Susan Berry, MD
Recruiting
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Margo Breilyn, MD
Recruiting
Case Western Medical College
Cleveland, Ohio, United States
Laura Konczal, MDLori-Anne Schillaci, MDRachel Tangen, PhD
Recruiting
Oregon Health and Science University
Portland, Oregon, United States
Cary Harding, MD
Recruiting
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Can Ficicioglu, MD
Recruiting
Baylor College of Medicine
Houston, Texas, United States
Sandesh Nagamani, MD
Recruiting
Children'S Hospital and Regional Medical Center
Seattle, Washington, United States
Christina Lam, MD, PhD
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 28 Feb 2006
Primary completion: 31 Jul 2025
Completion: 31 Jul 2026
Last update posted: 13 Feb 2024
Registered enrollment: 1,500
Registry id: NCT00237315

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.