- Conditions
- Leber Hereditary Optic Neuropathy (LHON)
- Interventions
- NR082 Injection, Injection needle
- Drug · Device
- Lead sponsor
- Neurophth Therapeutics Inc
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Aurora, Colorado • Philadelphia, Pennsylvania
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Showing 25–48
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- Conditions
- Age-related Macular Degeneration
- Interventions
- CNTO 2476, iTrack Model 275 micro catheter
- Biological · Procedure
- Lead sponsor
- Janssen Research & Development, LLC
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2017
- U.S. locations
- 2
- States / cities
- Arcadia, California • Philadelphia, Pennsylvania
- Conditions
- Dry Macular Degeneration
- Interventions
- SS-OCT imaging
- Device
- Lead sponsor
- Boston Image Reading Center
- Industry
- Eligibility
- Not listed
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Miami, Florida • Boston, Massachusetts + 1 more
- Conditions
- Leber's Hereditary Optic Neuropathy (LHON)
- Interventions
- Idebenone
- Drug
- Lead sponsor
- Santhera Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 199 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2021
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more
- Conditions
- Geographic Atrophy, Macular Degeneration
- Interventions
- Avacincaptad pegol (ACP)
- Drug
- Lead sponsor
- Astellas Pharma Global Development, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 65
- States / cities
- Phoenix, Arizona • Scottsdale, Arizona • Encino, California + 59 more
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Interventions
- Not listed
- Lead sponsor
- PYC Therapeutics
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 2
- States / cities
- Miami, Florida • Seattle, Washington
- Conditions
- Age-Related Macular Degeneration
- Interventions
- Minocycline
- Drug
- Lead sponsor
- National Eye Institute (NEI)
- NIH
- Eligibility
- 55 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2023
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Optic, Atrophy, Hereditary, Leber
- Interventions
- GS010, Sham Intravitreal Injection
- Biological · Device
- Lead sponsor
- GenSight Biologics
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- Not listed
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2037
- U.S. locations
- 1
- States / cities
- Columbia, Maryland
- Conditions
- PSP, PSP - Progressive Supranuclear Palsy, Corticobasal Syndrome, Corticobasal Syndrome(CBS), Corticobasal Degeneration Syndrome, Corticobasal Degeneration, Corticobasal Degeneration (CBD), Corticobasal Syndrome (CBS), MSA, MSA - Multiple System Atrophy, MSA-C, Multiple System Atrophy, Multiple System Atrophy (MSA) With Orthostatic Hypotension, Multiple System Atrophy - Cerebellar Subtype (MSA-C), Multiple System Atrophy - Parkinsonian Subtype (MSA-P), Multiple System Atrophy, Cerebellar Type, Multiple System Atrophy, Parkinsonian Type, Progressive Supranuclear Palsy, Progressive Supranuclear Palsy(PSP), Progressive Supranuclear Palsy (PSP)
- Interventions
- Whole genome sequencing will be performed at the NIH
- Other
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 35 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Frontotemporal Degeneration (FTD), ALS (Amyotrophic Lateral Sclerosis), Primary Progressive Aphasia (PPA), Familial Frontotemporal Lobar Degeneration (fFTLD), Fronto-temporal Lobar Dementia, Dementia With Lewy Bodies (DLB), Cortical Basal Syndrome (CBS), Alzheimer's Disease (AD), Progressive Supranuclear Palsy(PSP), Parkinson's Disease (PD), Parkinson's Disease Dementia (PDD), Limbic-predominant Age-related TDP-43 Encephalopathy (LATE), Posterior Cortical Atrophy (PCA), Primary Lateral Sclerosis (PLS), Primary Muscular Atrophy (PMA)
- Interventions
- Not listed
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2070
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Leber Hereditary Optic Neuropathy
- Interventions
- GS010, Placebo
- Genetic · Drug
- Lead sponsor
- GenSight Biologics
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 98 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 7
- States / cities
- Pasadena, California • Aurora, Colorado • Atlanta, Georgia + 4 more
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy
- Interventions
- Surgical implantation of the CPCB-RPE1 implant
- Device
- Lead sponsor
- Regenerative Patch Technologies, LLC
- Industry
- Eligibility
- 55 Years to 90 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2040
- U.S. locations
- 5
- States / cities
- Beverly Hills, California • Huntington Beach, California • Los Angeles, California + 2 more
- 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
- Conditions
- Optic Neuropathy
- Interventions
- magnetic resonance image (MRI), Optical Coherence Tomography (OCT)
- Device
- Lead sponsor
- University of Miami
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2018
- U.S. locations
- 1
- States / cities
- Miami, Florida
- Conditions
- Wolfram Syndrome
- Interventions
- AMX0035
- Drug
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Industry
- Eligibility
- 17 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Geographic Atrophy
- Interventions
- OpRegen
- Biological
- Lead sponsor
- Genentech, Inc.
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 15
- States / cities
- Beverly Hills, California • Sacramento, California • San Francisco, California + 12 more
- Conditions
- Geographic Atrophy
- Interventions
- No intervention
- Other
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 296 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 23
- States / cities
- Springdale, Arkansas • Campbell, California • Glendale, California + 19 more
- Conditions
- Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
- Interventions
- Mutation analysis
- Genetic
- Lead sponsor
- State University of New York at Buffalo
- Other
- Eligibility
- 0 Years to 65 Years
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration, Retinal Disease, Retinal Degeneration
- Interventions
- GEM103
- Drug
- Lead sponsor
- Gemini Therapeutics, Inc.
- Industry
- Eligibility
- 50 Years and older
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 29
- States / cities
- Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 26 more
- Conditions
- Stargardt Disease
- Interventions
- Not listed
- Lead sponsor
- AAVantgarde Bio Srl
- Industry
- Eligibility
- 8 Years to 50 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Stargardt Disease
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 259 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 6
- States / cities
- Baltimore, Maryland • Cleveland, Ohio • Philadelphia, Pennsylvania + 2 more
- Conditions
- Leber's Hereditary Optic Neuropathy
- Interventions
- EPI-743
- Drug
- Lead sponsor
- Edison Pharmaceuticals Inc
- Industry
- Eligibility
- 8 Years to 65 Years
- U.S. locations
- 1
- States / cities
- Los Angeles, California