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ClinicalTrials.gov public records Last synced May 21, 2026, 8:12 PM 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 Depletion Disorder Clear all
Completed No phase listed Observational
View directory record Official record
Conditions
Thymidine Kinase 2 Deficiency
Interventions
Observational retrospective data
Other
Lead sponsor
Zogenix MDS, Inc.
Industry
Eligibility
Not listed
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Indianapolis, Indiana • Akron, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced May 21, 2026, 8:12 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced May 21, 2026, 8:12 PM 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 21, 2026, 8:12 PM 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 21, 2026, 8:12 PM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
San Diego, California • Atlanta, Georgia • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced May 21, 2026, 8:12 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Thymidine Kinase 2 Deficiency
Interventions
doxecitine and doxribtimine
Drug
Lead sponsor
UCB BIOSCIENCES, Inc.
Industry
Eligibility
Not listed
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced May 21, 2026, 8:12 PM EDT