Longitudinal Study of Urea Cycle Disorders

NCT00237315 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 1500

Last updated 2024-02-13

No results posted yet for this study

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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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00237315 on ClinicalTrials.gov