- Conditions
- Leber Hereditary Optic Neuropathy (LHON)
- Interventions
- NFS-02 Injection
- Drug
- Lead sponsor
- Neurophth Therapeutics Inc
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
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 817–840
of 1,148
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
- Interventions
- ALD-101
- Biological
- Lead sponsor
- Aldagen
- Industry
- Eligibility
- Up to 16 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 3
- States / cities
- Los Angeles, California • New York, New York • Durham, North Carolina
- Conditions
- Epigastric Pain, Retrosternal Pain, Esophagitis, Gastrointestinal Symptoms
- Interventions
- Not listed
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- 5 Years to 17 Years
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2015
- U.S. locations
- 1
- States / cities
- Milwaukee, Wisconsin
- Conditions
- Tourette Syndrome, Persistent Tic Disorder
- Interventions
- Mindfulness-based Intervention for Tics, Tic Information and Coping Strategies
- Behavioral
- Lead sponsor
- Bowdoin College
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 2
- States / cities
- Brunswick, Maine • Boston, Massachusetts
- Conditions
- CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
- Interventions
- scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
- Drug
- Lead sponsor
- The Charlotte and Gwenyth Gray Foundation
- Other
- Eligibility
- 4 Months and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Hereditary Spastic Paraplegia
- Interventions
- D-cycloserine
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 6 Years to 6 Years · Female only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
- Interventions
- DUOC-01
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 1 Week to 22 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Wilson Disease
- Interventions
- Not listed
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Miami, Florida • Chicago, Illinois + 2 more
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 236 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Huntington Disease
- Interventions
- Fenofibrate, Placebo
- Drug
- Lead sponsor
- University of California, Irvine
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 1
- States / cities
- Irvine, California
- Conditions
- Neurofibromatosis 1, Nerve Sheath Neoplasms
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 3 Years to 120 Years
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2035
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Friedreich Ataxia, Cardiomyopathy
- Interventions
- Not listed
- Lead sponsor
- Lexeo Therapeutics
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 8
- States / cities
- La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
- Conditions
- Neurofibromatosis 2
- Interventions
- Penetrating auditory brainstem implant
- Device
- Lead sponsor
- FDA Office of Orphan Products Development
- Federal
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2003
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Pasadena, California • Englewood, Colorado
- Conditions
- Friedreich Ataxia
- Interventions
- Idebenone
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, SPG4, SPG5A, Spastic Paraplegia 4, Spastic Paraplegia 5A, Early Onset Hereditary Spastic Paraplegia, Neuromuscular Diseases, Spastic Paraplegia, Hereditary
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 11
- States / cities
- Miami, Florida • Iowa City, Iowa • Boston, Massachusetts + 6 more
- Conditions
- Pantothenate Kinase-associated Neurodegeneration (PKAN)
- Interventions
- Not listed
- Lead sponsor
- Susan J. Hayflick
- Other
- Eligibility
- 7 Years to 75 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2018
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Metastatic Castration-resistant Prostate Cancer
- Interventions
- BMS-986365, Enzalutamide, Abiraterone, Docetaxel, Predinsone/Prednisolone
- Drug
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 960 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 48
- States / cities
- Birmingham, Alabama • Gilbert, Arizona • Anaheim, California + 34 more
- Conditions
- Healthy
- Interventions
- Neurofeedback treatment, At-home therapy
- Device · Behavioral
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Wilson Disease
- Interventions
- Standard of Care Medications
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Chicago, Illinois • Ann Arbor, Michigan + 2 more
- Conditions
- Alzheimer's Disease, Huntington's Disease
- Interventions
- Dimebon
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009
- U.S. locations
- 2
- States / cities
- Miami, Florida • Saint Paul, Minnesota
- Conditions
- Physical Disability
- Interventions
- EnhanceWellness for Disability (EW-D), Wellness Education
- Behavioral
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 45 Years to 64 Years
- Enrollment
- 516 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- Conditions
- Tuberous Sclerosis Complex
- Interventions
- Ganaxolone, Placebo
- Drug
- Lead sponsor
- Marinus Pharmaceuticals
- Industry
- Eligibility
- 1 Year to 65 Years
- Enrollment
- 129 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 23
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Orange, California + 20 more
- Conditions
- Fertility Issues
- Interventions
- Not listed
- Lead sponsor
- Bar-Chama, Natan, M.D.
- Other
- Eligibility
- 18 Years to 50 Years · Male only
- Enrollment
- 75 participants
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Cresskill, New Jersey