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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Congenital Lactic Acidosis Clear all
No Longer Available No phase listed Expanded access

Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access

NCT01797276
View directory record Official record
Conditions
Mitochondrial Enzyme Deficiencies
Interventions
Dichloroacetate
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 50 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 7, 2017 · Synced Jul 22, 2026, 10:22 AM EDT
Completed Phase 2 Interventional

A Phase 2b Study of Zagociguat in Patients With MELAS

NCT06402123
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg, zagociguat 30mg, Placebo
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 22, 2026, 10:22 AM EDT
Recruiting Phase 3 Interventional

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

NCT06451757
View directory record Official record
Conditions
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions
Sonlicromanol, Placebo
Drug
Lead sponsor
Khondrion BV
Industry
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 22, 2026, 10:22 AM EDT
Terminated Phase 2 Interventional Results available

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445
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 Jul 22, 2026, 10:22 AM EDT
Completed Phase 2 Interventional

Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia

NCT00004493
View directory record Official record
Conditions
Lactic Acidosis
Interventions
sodium dichloroacetate
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
3 Months and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Jul 22, 2026, 10:22 AM EDT
Active, not recruiting Phase 2 Interventional

An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

NCT06961344
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
Not listed
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
9
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 22, 2026, 10:22 AM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

NCT04475549
View directory record Official record
Conditions
MELAS
Interventions
IW-6463 Tablets
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2024 · Synced Jul 22, 2026, 10:22 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Pharmacotoxicology of Trichloroethylene Metabolites

NCT00874276
View directory record Official record
Conditions
Congenital Lactic Acidosis
Interventions
Dichloroacetate (DCA), Genetic Marker on Chromosome 14q24.3
Drug · Genetic
Lead sponsor
University of Florida
Other
Eligibility
21 Years to 50 Years
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2012
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 3, 2015 · Synced Jul 22, 2026, 10:22 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Dichloroacetate Kinetics, Metabolism and Toxicology

NCT00015015
View directory record Official record
Conditions
Lactic Acidosis
Interventions
Dichloroacetate
Drug
Lead sponsor
National Institute of Environmental Health Sciences (NIEHS)
NIH
Eligibility
3 Months to 65 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 3, 2006 · Synced Jul 22, 2026, 10:22 AM EDT
Completed No phase listed Observational

Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia

NCT00004353
View directory record Official record
Conditions
Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1978
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jul 9, 2008 · Synced Jul 22, 2026, 10:22 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

NCT01694940
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 Jul 22, 2026, 10:22 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

NCT06792500
View directory record Official record
Conditions
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
Glycerol Tributyrate
Drug
Lead sponsor
George Washington University
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jul 22, 2026, 10:22 AM EDT
Not listed Phase 2 Interventional

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

NCT00068913
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Dichloroacetate
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 22, 2026, 10:22 AM EDT
Completed Phase 2 Interventional Results available

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 25, 2016 · Synced Jul 22, 2026, 10:22 AM EDT
Completed No phase listed Observational

Lactate Acidosis in Postoperative Hearts

NCT03025659
View directory record Official record
Conditions
Post-cardiac Surgery, Hyperlactatemia
Interventions
Pyruvate levels
Other
Lead sponsor
University of Florida
Other
Eligibility
Up to 21 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 22, 2020 · Synced Jul 22, 2026, 10:22 AM EDT