The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) (NAHIM)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01694953|
Recruitment Status : Recruiting
First Posted : September 27, 2012
Last Update Posted : February 27, 2019
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.
|Condition or disease|
|Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)|
|Study Type :||Observational|
|Estimated Enrollment :||10 participants|
|Official Title:||The Rare Disease Clinical Research Network Natural History Study of MNGIE|
|Study Start Date :||February 2013|
|Estimated Primary Completion Date :||June 2020|
|Estimated Study Completion Date :||June 2020|
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.
- Timed Water Swallow [ Time Frame: 5 years ]The timed water swallow test evaluates the time it takes to swallow a small cup of water
- Degree of Neuropathy [ Time Frame: 5 years ]A general neurological exam will be performed to assess the degree of neuropathy.
- Score on Mini Mental State Exam [ Time Frame: 5 years ]A short assessment of cognitive function will be performed.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01694953
|Contact: Kris Engelstad, MS||212-305-6834||NAMDC@columbia.edu|
|United States, New York|
|New York, New York, United States, 10032|
|Contact: Kris Engelstad, MS 212-305-6834 email@example.com|
|Principal Investigator: Michio Hirano, MD|
|Principal Investigator:||Michio Hirano, MD||Columbia University|