Mitochondrial Neurogastrointestinal EncephalomyopathyNCT04245917
Natural History Study of MNGIE
An observational study of Mitochondrial Neurogastrointestinal Encephalomyopathy, run by Entrada Therapeutics, Inc. at 5 sites in 2 countries.
Trial summary
Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Mitochondrial Neurogastrointestinal Encephalomyopathy
- What is being tested?
- Non-interventional
- How many people
- 60 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 6 Aug 2020to Feb 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male or female MNGIE patients greater than 5 years of age
Who may be able to join
- Has MNGIE
Who cannot join
- Substance abuser
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
United States3 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Baylor St Luke's Medical Center and Texas Children's Hospital at Baylor College of Medicine
Houston, Texas, 77030
Columbia University
New York, New York, 10032
Turkey (Türkiye)2 sites
Gazi Üniversitesi Tıp Fakültesi
Ankara, 06500
Ege University Medical Faculty; Department of Pediatrics
Bornova, İzmir
About this study
Official title: Natural History Study of Mitochondrial Neurogastrointestinal Encephalomyopathy
Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
- Being tested
- Non-interventional
- Keywords
- MNGIE
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-interventionalOther
Non-interventional natural history study
| Study group | Role | What they receive |
|---|---|---|
| MNGIE Patients | Patients with mitochondrial neurogastrointestinal encephalomyopathy Receives: Other: Non-interventional |
What is being measured
The primary outcome is the main question the study is designed to answer.
MNGIE Clinical Course
To evaluate the clinical course of MNGIE patients
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2020
- Study start6 Aug 2020
- Primary completionFeb 2025 (estimated)
- Study completionFeb 2026 (estimated)
Why it stopped: Business Reason
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial Neurogastrointestinal EncephalomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Entrada Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michio Hirano, MD, Study chair, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04245917
- Sponsor's protocol ID
- ET-NHS-901
- Status last verified
- Jul 2021
- First submitted
- 27 Jan 2020
- Last updated
- 22 Jul 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study of Mitochondrial Neurogastrointestinal Encephalomyopathy
- ClinicalTrials.gov ID
- NCT04245917
- Acronym / sponsor ID
- ET-NHS-901
- Keywords
- MNGIE
- Study type
- Observational
- Recruitment status
- SuspendedReason stopped: Business Reason
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Entrada Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Aug 2020 (actual)
- Primary completion
- Feb 2025 (estimated)
- Study completion
- Feb 2026 (estimated)
- First posted
- 29 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MNGIE Patients | – | Other: Non-interventionalPatients with mitochondrial neurogastrointestinal encephalomyopathy |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male or female MNGIE patients greater than 5 years of age
Key inclusion criteria (1 listed)
- Has MNGIE
Key exclusion criteria (1 listed)
- Substance abuser
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-interventionalNon-interventional natural history study | Other | MNGIE Patients |
Primary outcome
MNGIE Clinical Course
Time frame: 5 years
To evaluate the clinical course of MNGIE patients
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2021.
- Individual participant dataSharing planned: No
Related trials for Mitochondrial Neurogastrointestinal Encephalomyopathy
RecruitingObservational study
MNGIE Natural History Study
MNGIE, Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)
RecruitingObservational study
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Mitochondrial Disorders, Mitochondrial Genetic Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michio Hirano, MD study chair
Columbia University
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mitochondrial Neurogastrointestinal Encephalomyopathy, run by Entrada Therapeutics, Inc. at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 22 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MNGIE Clinical Course (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2021.