- Conditions
- GM1 Gangliosidosis
- Interventions
- Natural history
- Other
- Lead sponsor
- LYSOGENE
- Industry
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Plymouth, Massachusetts
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 961–984
of 1,710
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- SRP-4045, SRP-4053, Placebo
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years to 13 Years · Male only
- Enrollment
- 228 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2025
- U.S. locations
- 23
- States / cities
- Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
- Conditions
- Mucolipidosis Type IV
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 1 Year to 64 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2021
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Advanced Solid Tumors
- Interventions
- Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
- Drug
- Lead sponsor
- Bayer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 8
- States / cities
- Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
- Conditions
- Stroke
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 65 Years to 90 Years · Female only
- Enrollment
- 1,426 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2007
- U.S. locations
- 1
- States / cities
- Winston-Salem, North Carolina
- Conditions
- Pantothenate Kinase-Associated Neurodegeneration
- Interventions
- Deferiprone oral solution, Placebo
- Drug
- Lead sponsor
- ApoPharma
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 89 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Cerebral Palsy, Arthrogryposis, Spinal Muscular Atrophy, Other Central Nervous System or Musculoskeletal Disorders
- Interventions
- Power mobility
- Other
- Lead sponsor
- University of Oklahoma
- Other
- Eligibility
- 14 Months to 30 Months
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 1
- States / cities
- Oklahoma City, Oklahoma
- Conditions
- Friedreich's Ataxia (FA)
- Interventions
- SGT-212
- Drug
- Lead sponsor
- Solid Biosciences Inc.
- Industry
- Eligibility
- 18 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
- Conditions
- Phenylketonuria (PKU) and Hyperphenylalaninemia
- Interventions
- PheCheck, Dried Blood Spot Cards.
- Device · Diagnostic Test
- Lead sponsor
- Aptatek BioSciences, Inc
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 2
- States / cities
- Chicago, Illinois • Boston, Massachusetts
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Head and Neck Cancer, Kidney Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
- Interventions
- filgrastim, temozolomide, peripheral blood stem cell transplantation
- Biological · Drug · Procedure
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2005
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Huntington Disease
- Interventions
- Transcranial direct current stimulation (tDCS), Sham tDCS
- Device
- Lead sponsor
- The University of Texas Health Science Center at San Antonio
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- San Antonio, Texas
- Conditions
- Tourette Syndrome, ADHD
- Interventions
- Modified Comprehensive Behavioral Intervention for Tics, Comprehensive Behavioral Intervention for Tics
- Behavioral
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 10 Years to 17 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Vascular; Lesion, Central Nervous System, Brain Tumor, Cerebral Aneurysm, Cerebral AVM
- Interventions
- GLOW800, indocyanine green (ICG)
- Device · Drug
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma, Glioma
- Interventions
- Selumetinib
- Drug
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 3 Years to 45 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- Up to 16 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency, Ceramidase Deficiency, N-Laurylsphingosine Deacylase Deficiency, ASAH1 Mutation
- Interventions
- Not listed
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 2
- States / cities
- Oakland, California • Washington D.C., District of Columbia
- Conditions
- Ataxia Telangiectasia, Genetic Syndrome
- Interventions
- EryDex System
- Combination Product
- Lead sponsor
- Quince Therapeutics S.p.A.
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
- Conditions
- Pelizaeus-Merzbacher Disease, PMD
- Interventions
- HuCNS-SC transplant in the lead-in phase
- Biological
- Lead sponsor
- StemCells, Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Fabry Disease
- Interventions
- Enzyme Replacement Therapy (ERT)
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 82 participants
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Friedreich's Ataxia
- Interventions
- Low dose cohort, High dose cohort
- Drug
- Lead sponsor
- Biojiva LLC
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 2
- States / cities
- Long Beach, California • Tampa, Florida
- Conditions
- Tourette's Disorder
- Interventions
- Wearable short wavelength light therapy
- Other
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 13 Years to 17 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Solid Tumors
- Interventions
- Trabectedin (ecteinascidin-743, ET-743, YONDELIS [R])
- Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 4 Years to 16 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 1 Year and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts