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ClinicalTrials.gov public records Last synced May 22, 2026, 12:13 AM EDT

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Showing 1–24 of 109 matching trials from the live ClinicalTrials.gov search.
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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 Mar 24, 2026 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Hypoxic-Ischemic Encephalopathy
Interventions
Optical Coherence Tomography
Device
Lead sponsor
Duke University
Other
Eligibility
Up to 20 Days
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
2
States / cities
Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 7, 2021 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Congenital Portosystemic Shunt, CPSS (Congenital Portosystemic Shunt)
Interventions
Shunt Closure
Procedure
Lead sponsor
Prof. Valérie Mc Lin
Other
Eligibility
1 Day and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
2
States / cities
New Haven, Connecticut • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 17, 2025 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Infant Development, Infant,Premature, Hydrocephalus, Neonatal Encephalopathy, Infarction, PVL, Thrombosis, Intraventricular Hemorrhage
Interventions
pacifier-activated device
Device
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
32 Weeks to 12 Months
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced May 22, 2026, 12:13 AM EDT
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 May 3, 2026 · Synced May 22, 2026, 12:13 AM EDT
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, 12:13 AM EDT
Conditions
Neonatal Encephalopathy, Hypoxic-Ischemic Encephalopathy Mild
Interventions
Darbepoetin Alfa, Normal Saline
Drug
Lead sponsor
University of New Mexico
Other
Eligibility
1 Hour to 24 Hours
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Hypothermia
Interventions
Not listed
Lead sponsor
Drexel University
Other
Eligibility
6 Hours to 24 Hours
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2017 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Infant, Newborn, Hypoxia-Ischemia, Brain
Interventions
Induced hypothermia, Control
Device
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
Up to 6 Hours
Enrollment
208 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
16
States / cities
Birmingham, Alabama • Palo Alto, California • New Haven, Connecticut + 13 more
Source: ClinicalTrials.gov public record
Updated Mar 21, 2019 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Acute Kidney Injury, Hypoxic-Ischemic Encephalopathy, Caffeine
Interventions
Caffeine citrate
Drug
Lead sponsor
University of Arkansas
Other
Eligibility
0 Hours to 24 Hours
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
2
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated May 9, 2024 · Synced May 22, 2026, 12:13 AM EDT
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Hypoxic Ischemic Encephalopathy of Newborn, Hypoxic Ischemic Encephalopathy, Hypoxic Ischemic Encephalopathy (HIE)
Interventions
Genome sequencing
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
0 Days to 1 Year
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 30, 2025 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Neonatal Seizure, Hypoxic-Ischemic Encephalopathy, Stroke, Intracranial Hemorrhages, Epilepsy, Cerebral Palsy, Intellectual Disability
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
2 Years to 8 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
9
States / cities
San Francisco, California • Stanford, California • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Hypoxic Ischemic Encephalopathy
Interventions
Olympic Cool Cap
Device
Lead sponsor
Vanderbilt University
Other
Eligibility
30 Minutes to 6 Hours
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 28, 2019 · Synced May 22, 2026, 12:13 AM EDT
Completed No phase listed Observational Results available
Conditions
Hypoxic-Ischemic Encephalopathy, Brain Injury, Neonatal Seizure
Interventions
Neurologic examination
Other
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Up to 6 Hours
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
4
States / cities
Detroit, Michigan • Columbus, Ohio • Providence, Rhode Island + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Mild Hypoxic Ischemic Encephalopathy of Newborn
Interventions
Normothermia, Whole body therapeutic hypothermia
Procedure
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
36 Weeks and older
Enrollment
460 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Cerebral Palsy, Hypoxic-Ischemic Encephalopathy
Interventions
Sensorimotor Intervention
Behavioral
Lead sponsor
University of Southern California
Other
Eligibility
Up to 6 Months
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Seizures
Interventions
Bumetanide, Normal Saline as Placebo
Drug
Lead sponsor
Soul, Janet , M.D.
Individual
Eligibility
Up to 44 Weeks
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
4
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 16, 2020 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
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, 12:13 AM EDT
Completed Not applicable Interventional Results available
Conditions
Encephalopathy, Birth Defect, Intellectual Disability, Multiple Congenital Anomaly, Metabolic Disease, Epilepsy, Neuro-Degenerative Disease, Cerebral Palsy, Developmental Delay, Developmental Defect
Interventions
Whole Exome Sequencing
Diagnostic Test
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 25 Years
Enrollment
529 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Fresno, California • Oakland, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 17, 2023 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Hypoxic-Ischemic Encephalopathy
Interventions
RLS-0071, Placebo
Drug
Lead sponsor
ReAlta Life Sciences, Inc.
Industry
Eligibility
Up to 10 Hours
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
15
States / cities
Little Rock, Arkansas • Orange, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced May 22, 2026, 12:13 AM EDT
Conditions
Neonatal Encephalopathy, Hypothermia Neonatal, Magnetic Resonance Spectroscopy
Interventions
Therapeutic hypothermia
Other
Lead sponsor
Thayyil, Sudhin
Individual
Eligibility
Up to 6 Hours
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
Michigan Center, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 3, 2023 · Synced May 22, 2026, 12:13 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers
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 22, 2026, 12:13 AM EDT