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ClinicalTrials.gov public records Last synced May 22, 2026, 3:54 AM EDT

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Showing 1–6 of 6 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) Clear all
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced May 22, 2026, 3:54 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 16, 2025 · Synced May 22, 2026, 3:54 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
6,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced May 22, 2026, 3:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced May 22, 2026, 3:54 AM EDT
Suspended No phase listed Observational
View directory record Official record
Conditions
Mitochondrial Neurogastrointestinal Encephalomyopathy
Interventions
Non-interventional
Other
Lead sponsor
Entrada Therapeutics, Inc.
Industry
Eligibility
5 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Aurora, Colorado • New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 21, 2021 · Synced May 22, 2026, 3:54 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 26, 2024 · Synced May 22, 2026, 3:54 AM EDT