- Conditions
- Developmental and Epileptic Encephalopathy
- Interventions
- LP352
- Drug
- Lead sponsor
- Longboard Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 66 Years
- Enrollment
- 324 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 20
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 16 more
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Showing 1–24
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- Conditions
- Perinatal Death, Respiratory Distress Syndrome, Newborn, Hypoxic-Ischemic Encephalopathy, Neonatal Seizure, Meconium Aspiration Syndrome, Intracranial Hemorrhages, Neonatal Hypotension
- Interventions
- Room air
- Other
- Lead sponsor
- Loma Linda University
- Other
- Eligibility
- 18 Years to 55 Years · Female only
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Loma Linda, California
- 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
- 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
- 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
- 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
- Conditions
- Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
- Interventions
- Infusion of autologous cord blood, Placebo
- Biological
- Lead sponsor
- Michael Cotten
- Other
- Eligibility
- 0 Hours to 6 Hours
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Hypoxic-Ischemic Encephalopathy
- Interventions
- Caffeine Citrate 5 mg/kg, Caffeine Citrate 10 mg/kg
- Drug
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- Up to 24 Hours
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- 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
- Conditions
- Down Syndrome, Regression
- Interventions
- Lorazepam, Intravenous immunoglobulin (IVIG), Tofacitinib
- Drug
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 8 Years to 30 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Aurora, Colorado
- Conditions
- Neonatal Encephalopathy
- Interventions
- Telemedicine consult
- Diagnostic Test
- Lead sponsor
- MaineHealth
- Other
- Eligibility
- 0 Hours to 6 Hours
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Portland, Maine
- 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
- Conditions
- Developmental Dysphasia, Epileptic Encephalopathy, Childhood-Onset, X-Linked Intellectual Disability
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 6 Years to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 1
- States / cities
- San Francisco, California
- 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
- 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
- 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
- Conditions
- Hypoxic Ischemic Encephalopathy
- Interventions
- blood sample
- Procedure
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 1 Day to 3 Days
- Enrollment
- 39 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2017
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- 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
- Conditions
- Neonatal Encephalopathy
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 6 Years to 8 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Infant, Newborn, Hypoxia, Brain, Hypoxia-Ischemia, Brain, Encephalopathy, Hypoxic-Ischemic, Hypoxic-Ischemic Encephalopathy, Ischemic-Hypoxic Encephalopathy
- Interventions
- Hypothermia, Normothermic Control
- Procedure
- Lead sponsor
- NICHD Neonatal Research Network
- Network
- Eligibility
- 6 Hours to 24 Hours
- Enrollment
- 168 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2016
- U.S. locations
- 22
- States / cities
- Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 18 more
- Conditions
- Hypoxic-Ischemic Encephalopathy, Brain Ischemia Hypoxia
- Interventions
- Sulfur hexafluoride lipid-type A microspheres
- Drug
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 1 Minute to 18 Months
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial, Mitochondrial Disorders, Leigh Disease
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 4 Weeks to 115 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Seizures, Encephalopathy, Asphyxiated Newborn
- Interventions
- Not listed
- Lead sponsor
- Sharp HealthCare
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2015
- U.S. locations
- 1
- States / cities
- San Diego, California
- 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