Longitudinal Study of Mitochondrial Hepatopathies
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Conditions studied: Acute Liver Failure, Mitochondrial Diseases, End Stage Liver Disease, Respiratory Chain Deficiencies, Mitochondrial, Disorder of Fatty Acid Oxidation
In brief
The specific aims of this study are (1) to determine the clinical phenotypes and natural history of hepatic RC and FAO disorders, (2) to determine the correlation between genotype and phenotype, (3) to determine if circulating biomarkers reflect diagnosis and predict liver disease progression and survival with the native liver, (4) to determine the clinical outcome of these disorders following liver transplantation, and (5) to develop a repository of serum, plasma, urine, tissue and DNA specimens that will be used in ancillary studies. To accomplish these aims, the ChiLDReN investigators at clinical sites (currently 9 sites) will prospectively collect defined data and specimens in a uniform fashion at fixed intervals in a relatively large number of subjects. Clinical information collected from subjects and their parents will enhance the potential for meaningful research in these disorders. A biobank of previously collected subject specimens and DNA samples will be established for use in ancillary studies to be performed in addition to this study.
Key facts
- Study ID
- NCT01148550
- Run by
- Arbor Research Collaborative for Health
- People needed
- 90
- Starts
- 2010-08-18
- Expected to finish
- 2029-05-31
- Last updated by the study team
- 2026-06-04
Who can join
Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Children's Hospital Los Angeles — Los Angeles, California, United States
- University of California at San Francisco (UCSF) — San Francisco, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Children's Healthcare of Atlanta - Emory University — Atlanta, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital — Chicago, Illinois, United States
- Riley Hospital for Children — Indianapolis, Indiana, United States
- Johns Hopkins School of Medicine — Baltimore, Maryland, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Mount Sinai Medical Center — New York, New York, United States
- Children's Hospital Medical Center — Cincinnati, Ohio, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Texas Children's Hospital (Baylor College of Medicine) — Houston, Texas, United States
- University of Utah — Salt Lake City, Utah, United States
- Seattle Children's Hospital — Seattle, Washington, United States
- Hospital for Sick Children — Toronto, Ontario, Canada
Full record on ClinicalTrials.gov
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