The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)

(NAHIM Trial)

Recruiting in Palo Alto (17 mi)

Overseen by Michio Hirano, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: Columbia University

No Placebo Group

Trial Summary

What is the purpose of this trial?

This is a multi-center natural history study of Mitochondrial NeurogastroIntestinal Encephalopathy (MNGIE). Patients will be followed over time to assess clinical symptoms. The investigators hope to learn more about the disease of MNGIE as well as develop useful measures of disease status for use in future clinical trials. Additional clinical centers will be listed as they become available.

Eligibility Criteria

Inclusion Criteria

homozygous or compound heterozygous mutations in the TYMP gene, and/or TP enzyme activity of <20% of normal.

Increased plasma Thd > 3 micromole/L

Increased plasma dUrd > 7.5 micromole/L

Participant Groups

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Columbia University New York, NY

Who Is Running the Clinical Trial?

Columbia University Lead Sponsor

National Institute of Neurological Disorders and Stroke (NINDS) Collaborator

References