Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 17, 2026, 9:23 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Developmental and Epileptic Encephalopathy
Interventions
gene therapy
Drug
Lead sponsor
Capsida Biotherapeutics, Inc.
Industry
Eligibility
18 Months to 7 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
4
States / cities
Aurora, Colorado • New York, New York • Philadelphia, Pennsylvania + 1 more
Conditions
Neonatal Encephalopathy
Interventions
Sildenafil, Ora-Blend
Drug
Lead sponsor
Pia Wintermark
Other
Eligibility
0 Minutes to 48 Hours
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Denver, Colorado
Conditions
Dravet Syndrome
Interventions
zorevunersen, Sham Comparator
Drug · Other
Lead sponsor
Stoke Therapeutics, Inc
Industry
Eligibility
2 Years to 17 Years
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
28
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 23 more
Conditions
Developmental and Epileptic Encephalopathy
Interventions
LP352, Placebo
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
31
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 23 more
Conditions
Newborn Hypoxic Ischemic Encephalopathy
Interventions
Melatonin, placebo
Drug
Lead sponsor
St. Louis University
Other
Eligibility
1 Hour to 8 Hours
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
St Louis, Missouri
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
Conditions
Encephalopathy, Radiation Effect
Interventions
Gocovri (extended-release amantidine)
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New York, New York
Conditions
Hypoxic-ischemic Encephalopathy
Interventions
Dexmedetomidine
Drug
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
1 Hour to 1 Day
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Epileptic Encephalopathy, SCN2A Encephalopathy
Interventions
1mg elsunersen, 0.5mg elsunersen
Drug
Lead sponsor
Praxis Precision Medicines
Industry
Eligibility
1 Day to 18 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
6
States / cities
San Diego, California • Aurora, Colorado • Chicago, Illinois + 3 more
Conditions
Epilepsy, Epilepsy in Children, Epilepsy; Seizure, Disease, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Epileptic Syndromes
Interventions
XEN496
Drug
Lead sponsor
Xenon Pharmaceuticals Inc.
Industry
Eligibility
1 Month to 6 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Tacoma, Washington
Conditions
Alzheimer Dementia, Alzheimer Disease, Delirium Confusional State, Encephalopathy, Cognitive Abnormality
Interventions
Quest AD-Detect blood test
Diagnostic Test
Lead sponsor
Covenant Health, US
Other
Eligibility
60 Years to 90 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Crossville, Tennessee • Harriman, Tennessee • Knoxville, Tennessee + 3 more
Conditions
Encephalopathy, Developmental Delay and Behavioral Changes, Cord Tumor Compression or Instability, Cord Tethering or Malformation
Interventions
Intermittent Propofol Sedation, Continuous Propofol Sedation
Procedure
Lead sponsor
Corewell Health West
Other
Eligibility
1 Month to 18 Years
Enrollment
276 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Grand Rapids, Michigan
Conditions
Developmental and Epileptic Encephalopathies, Epileptic Spasms, Genetic Epilepsy, Neonatal and Infant Epilepsy
Interventions
Ketogenic diet
Drug
Lead sponsor
Heather Olson
Other
Eligibility
0 Days to 9 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Enrolling by invitation No phase listed Observational

Florida Neonatal Neurologic Network

NCT02260271
Conditions
Hypoxic-Ischemic Encephalopathy
Interventions
Database Entry/Biospecimen Collection
Other
Lead sponsor
University of Florida
Other
Eligibility
1 Day to 3 Weeks
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2045
U.S. locations
1
States / cities
Gainesville, Florida
Completed Phase 2 Interventional Results available

CJD (Creutzfeldt-Jakob Disease) Quinacrine Study

NCT00183092
Conditions
Creutzfeldt-Jakob Disease
Interventions
Quinacrine, Placebo
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years and older
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
San Francisco, California
Conditions
Neonatal Hypoxic-Ischemic Encephalopathy (HIE)
Interventions
Cool-Cap
Device
Lead sponsor
Olympic Medical
Industry
Eligibility
1 Hour to 6 Hours
Enrollment
235 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2003
U.S. locations
19
States / cities
Little Rock, Arkansas • Oakland, California • San Diego, California + 15 more
Completed Not applicable Interventional Accepts healthy volunteers

Breath and Blood Ammonia Response to an Oral Protein Challenge

NCT02650245
Conditions
Cirrhosis
Interventions
EAS Myoplex Protein Drink + 10gm lactulose
Dietary Supplement
Lead sponsor
St. Luke's Hospital and Health Network, Pennsylvania
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Bethlehem, Pennsylvania
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
Conditions
Neonatal Encephalopathy
Interventions
Enteral Feeding
Procedure
Lead sponsor
NYU Langone Health
Other
Eligibility
0 Days to 2 Days
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
New York, New York
Completed Phase 2 Interventional Accepts healthy volunteers

Brain Imaging and Retreatment Study of Persistent Lyme Disease

NCT00037479
Conditions
Lyme Disease, Lyme Neuroborreliosis
Interventions
ceftriaxone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 65 Years
Enrollment
65 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
New York, New York
Conditions
Fatigue Syndrome, Chronic
Interventions
Naltrexone HCl
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 65 Years · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder), ACTL6B, ADNP, AHDC1, ANK2, ANKRD11, ARID1B, ASH1L, BCL11A, CHAMP1, CHD2, CHD8, CSNK2A1, CTBP1, CTNNB1 Gene Mutation, CUL3, DDX3X, DNMT3A, DSCAM, DYRK1A, FOXP1, GRIN2A, GRIN2B, HIVEP2-Related Intellectual Disability, HNRNPH2, KATNAL2, KDM5B, KDM6B, KMT2C Gene Mutation, KMT2E, KMT5B, MBD5, MED13L, PACS1, PPP2R5D-Related Intellectual Disability, PTCHD1, REST, SCN2A Encephalopathy, SETBP1 Gene Mutation, SETD5, SMARCA4 Gene Mutation, SMARCC2, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability, TBR1, ARHGEF9, HNRNPU, PPP3CA, PPP2R1A, SLC6A1, 2p16.3 Deletions, 5q35 Deletions, 5q35 Duplications, 7q11.23 Duplications, 15Q13.3 Deletion Syndrome, 16p11.2 Triplications, 16P12.2 Microdeletion, 16P13.11 Microdeletion Syndrome (Disorder), 17Q12 Microdeletion Syndrome (Disorder), 17Q12 Duplication Syndrome, 17Q21.31 Deletion Syndrome, 17q21.3 Duplications, ACTB, ADSL, AFF2, ALDH5A1, ANK3, ARX, ATRX Gene Mutation, AUTS2 Syndrome, BCKDK, BRSK2, CACNA1C, CAPRIN1, CASK, CASZ1, CHD3, CIC, CNOT3, CREBBP Gene Mutation, CSDE1, CTCF, DEAF1, DHCR7, DLG4, EBF3, EHMT1, EP300 Gene Mutation, GIGYF1, GRIN1, GRIN2D, IQSEC2-Related Syndromic Intellectual Disability, IRF2BPL, KANSL1, KCNB1, KDM3B, NEXMIF, KMT2A, MBOAT7, MEIS2, MYT1L, NAA15, NBEA, NCKAP1, NIPBL, NLGN2, NLGN3, NLGN4X, NR4A2, NRXN1, NRXN2, NSD1 Gene Mutation, PHF21A, PHF3, PHIP, PSMD12, RELN, RERE, RFX3, RIMS1, RORB, SCN1A, SETD2 Gene Mutation, SHANK2, SIN3A, SLC9A6, SON, SOX5, SPAST, SRCAP, TAOK1, TANC2, TCF20, TLK2, TRIO, TRIP12, UPF3B, USP9X, VPS13B, WAC, WDFY3, ZBTB20, ZNF292, ZNF462, 2Q37 Deletion Syndrome, 9q34 Duplications, 15q15 Deletions, 15Q24 Deletion, NR3C2, SYNCRIP, 2q37.3 Deletion, 6q16 Deletion, 15q11.2 BP1-BP2 Deletion, 16p13.3 Deletion, 17Q11.2 Microduplication Syndrome (Disorder), 17p13.3, Xq28 Duplication, CLCN4, CSNK2B, DYNC1H1, EIF3F, GNB1, MED13, MEF2C, RALGAPB, SCN1B, YY1, Xp11.22 Duplication, PACS2, MAOA, MAOB, HNRNPC, HNRNPD, HNRNPK, HNRNPR, HNRNPUL2, 5P Deletion Syndrome, TCF7L2 Gene Mutation, HECW2, PPM1D, RNU4-2, SNAP25, FOXP2, ITSN1
Interventions
Not listed
Lead sponsor
Simons Searchlight
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2050
U.S. locations
2
States / cities
Boston, Massachusetts • Lewisburg, Pennsylvania
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
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
Completed No phase listed Observational Accepts healthy volunteers

Amyloid and Tauopathy PET Imaging in Acute and Chronic Traumatic Brain Injury

NCT02266563
Conditions
Traumatic Brain Injury, Chronic Traumatic Encephalopathy, Mild Cognitive Impairment
Interventions
Amyvid PET Scan, T807 PET scan
Drug
Lead sponsor
Samuel Gandy
Other
Eligibility
40 Years to 85 Years · Male only
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
New York, New York