Longitudinal Study of Mitochondrial Hepatopathies

(MITOHEP Trial)

Recruiting in Palo Alto (17 mi)

+15 other locations

Overseen by John C Magee, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Arbor Research Collaborative for Health

Stay on Your Current Meds

No Placebo Group

Trial Summary

What is the purpose of this trial?

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 15 sites) will prospectively collect defined data and specimens in a uniform fashion at fixed intervals in a relatively large number of subjects. Clinical information and DNA samples to be collected from subjects and their parents will enhance the potential for meaningful research in these disorders. A biobank of subject specimens and DNA samples will be established for use in ancillary studies to be performed in addition to this study.

Research Team

Ed Doo, MD

Principal Investigator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Ronald J Sokol, MD

Principal Investigator

University of Colorado, Denver

Lisa Henn, PhD

Principal Investigator

Arbor Research Collaborative for Health - Data Coordinating Center

John C Magee, MD

Principal Investigator

University of Michigan

Averell Sherker, MD

Principal Investigator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Eligibility Criteria

Inclusion Criteria

Children and young adults with suspected or documented hepatic RC defector FAO defect from birth to 18 years old (through 18 years).

Both genders, all races and ethnic groups.

Clinical Criteria 1 (any one of the following)

Treatment Details

Interventions

• Observational Study (Other)

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Hospital for Sick Children Toronto, Canada

University of Utah Salt Lake City, UT

Johns Hopkins School of Medicine Baltimore, MD

Washington University School of Medicine Saint Louis, MO

More Trial Locations

Who Is Running the Clinical Trial?

Arbor Research Collaborative for Health

Lead Sponsor

Trials

21

Patients Recruited

35,500+

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborator

Trials

2513

Patients Recruited

4,366,000+

University of Michigan

Collaborator

Trials

1891

Patients Recruited

6,458,000+