The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)

Part of paid clinical trials in New York, New York.

Sponsor
Columbia University
Study ID
NCT01694953
Status
Recruiting

Conditions

  • Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)

Eligibility Criteria

Sex
ALL
Age
5 Years - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Feb 28, 2013
Status verified
Apr 2025
Primary completion
Dec 31, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
20 participants (estimated)

Arms

  • Arm: Patients with MNGIE
    Patients of all races of any gender who are at least 5 years of age with a defect in thymidine phosphorylase may participate in this natural history study.

Primary Outcome Measure

Timed Water Swallow [ Time Frame: 5 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Columbia UniversityNew YorkNew York10032
Kris Engelstad, MS
212-305-6834
Michio Hirano, MD (PRINCIPAL_INVESTIGATOR)

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