Natural History Study of MNGIE

July 16, 2021 updated by: Entrada Therapeutics, Inc.

Natural History Study of Mitochondrial Neurogastrointestinal Encephalomyopathy

Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)

Study Overview

Status

Suspended

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Turkey
      • Ankara, Turkey, 06500
        • Gazi Üniversitesi Tıp Fakültesi
    • Izmir
      • Bornova, Izmir, Turkey
        • Ege University Medical Faculty; Department of Pediatrics
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • New York
      • New York, New York, United States, 10032
        • Columbia University
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor St Luke's Medical Center and Texas Children's Hospital at Baylor College of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Male or female MNGIE patients greater than 5 years of age

Description

Inclusion Criteria:

  • Has MNGIE

Exclusion Criteria:

  • Substance abuser

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
MNGIE Patients
Patients with mitochondrial neurogastrointestinal encephalomyopathy
Other: Non-interventional
Non-interventional natural history study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
MNGIE Clinical Course
Time Frame: 5 years
To evaluate the clinical course of MNGIE patients
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Entrada Therapeutics, Inc.

Investigators

  • Study Chair: Michio Hirano, MD, Columbia University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 6, 2020

Primary Completion (Anticipated)

February 1, 2025

Study Completion (Anticipated)

February 1, 2026

Study Registration Dates

First Submitted

January 27, 2020

First Submitted That Met QC Criteria

January 27, 2020

First Posted (Actual)

January 29, 2020

Study Record Updates

Last Update Posted (Actual)

July 22, 2021

Last Update Submitted That Met QC Criteria

July 16, 2021

Last Verified

July 1, 2021

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • ET-NHS-901

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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