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Natural History Study of MNGIE

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. Identifier: NCT04245917
Recruitment Status : Recruiting
First Posted : January 29, 2020
Last Update Posted : July 22, 2020
Information provided by (Responsible Party):
Entrada Therapeutics, Inc.

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

Condition or disease Intervention/treatment
Mitochondrial Neurogastrointestinal Encephalomyopathy Other: Non-interventional

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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History Study of Mitochondrial Neurogastrointestinal Encephalomyopathy
Estimated Study Start Date : July 21, 2020
Estimated Primary Completion Date : February 2025
Estimated Study Completion Date : February 2026

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

Primary Outcome Measures :
  1. MNGIE Clinical Course [ Time Frame: 5 years ]
    To evaluate the clinical course of MNGIE patients

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Male or female MNGIE patients greater than 5 years of age

Inclusion Criteria:

  • Has MNGIE

Exclusion Criteria:

  • Substance abuser

Information from the National Library of Medicine

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 identifier (NCT number): NCT04245917

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Contact: Catherine O'Brien 646-322-1104

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United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Amandla L Atilano-Roque, PhD    720-777-5480   
Principal Investigator: Austin Larson, MD         
United States, New York
Columbia University Recruiting
New York, New York, United States, 10032
Contact: Kris Engelstad, MS CGC    212-342-5767    KE4@CUMC.COLUMBIA.EDU   
Principal Investigator: Michio Hirano, MD         
United States, Texas
Baylor St Luke's Medical Center and Texas Children's Hospital at Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: May Ali, Clinical Research Coordinator, MD    832-822-4280 ext 832-822-1630   
Contact: Fernando Scaglia, MD         
Principal Investigator: Fernando Scaglia, MD         
Ege University Medical Faculty; Department of Pediatrics Recruiting
Bornova, Izmir, Turkey
Contact: Prof. Dr. Sema Kalkan Uçar    +90.232.390.1037   
Principal Investigator: Prof. Dr. Sema Kalkan Uçar         
Gazi Üniversitesi Tıp Fakültesi Recruiting
Ankara, Turkey, 06500
Contact: Fatih Ezgu, MD    +90-532-6853697   
Principal Investigator: Fatih Ezgu, MD         
Sponsors and Collaborators
Entrada Therapeutics, Inc.
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Study Chair: Michio Hirano, MD Columbia University
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Responsible Party: Entrada Therapeutics, Inc. Identifier: NCT04245917    
Other Study ID Numbers: ET-NHS-901
First Posted: January 29, 2020    Key Record Dates
Last Update Posted: July 22, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Entrada Therapeutics, Inc.: