NCT04245917

Brief Summary

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

Trial Health

37
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Trial recruitment is currently suspended
Enrollment
60

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Aug 2020

Longer than P75 for all trials

Geographic Reach
2 countries

5 active sites

Status
suspended

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 27, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 29, 2020

Completed
6 months until next milestone

Study Start

First participant enrolled

August 6, 2020

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2025

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2026

Completed
Last Updated

July 22, 2021

Status Verified

July 1, 2021

Enrollment Period

4.5 years

First QC Date

January 27, 2020

Last Update Submit

July 16, 2021

Conditions

Mitochondrial Neurogastrointestinal Encephalomyopathy

Keywords

MNGIE

Outcome Measures

Primary Outcomes (1)

  • MNGIE Clinical Course

    To evaluate the clinical course of MNGIE patients

    5 years

Study Arms (1)

MNGIE Patients

Patients with mitochondrial neurogastrointestinal encephalomyopathy

Other: Non-interventional

Interventions

Non-interventionalOTHER

Non-interventional natural history study

MNGIE Patients

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Male or female MNGIE patients greater than 5 years of age

You may qualify if:

  • Has MNGIE

You may not qualify if:

  • Substance abuser

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Baylor St Luke's Medical Center and Texas Children's Hospital at Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Ege University Medical Faculty; Department of Pediatrics

Bornova, İzmir, Turkey (Türkiye)

Location

Gazi Üniversitesi Tıp Fakültesi

Ankara, 06500, Turkey (Türkiye)

Location

Study Officials

  • Michio Hirano, MD

    Columbia University

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 27, 2020

First Posted

January 29, 2020

Study Start

August 6, 2020

Primary Completion

February 1, 2025

Study Completion

February 1, 2026

Last Updated

July 22, 2021

Record last verified: 2021-07

Data Sharing

IPD Sharing
Will not share

Locations

US(3)TU(2)