Trials
375 trials
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Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity
Recruiting now Phase 4 Drug / neuroprotection
Tom Hühne. Germany
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Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Recruiting now Phase 3 RNA / antisense
Laboratoires Thea. Gene: CEP290. Australia, Belgium, Brazil, Canada and 6 more
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A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Recruiting now Phase 3 Drug / neuroprotection
Nacuity Pharmaceuticals, Inc.. Not gene-specific. United States
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Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)
Recruiting now Phase 2 / Phase 3 Optogenetics, Gene therapy
Ray Therapeutics, Inc.. Not gene-specific. United States
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Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
Recruiting now Phase 1 / Phase 2 Gene therapy
AAVantgarde Bio Srl. Gene: MYO7A. Italy, United Kingdom
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Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies
Recruiting now Phase 1 / Phase 2 Cell therapy
Ace Cells Lab Limited. Egypt
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Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Recruiting now Phase 1 / Phase 2 Gene editing, Gene therapy
Ascidian Therapeutics, Inc. United States
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BS01 in Patients With Retinitis Pigmentosa
Recruiting now Phase 1 / Phase 2 Optogenetics, Gene therapy
Bionic Sight LLC. United States
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A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Recruiting now Phase 1 / Phase 2 Gene therapy, Cell therapy
BlueRock Therapeutics. United States
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Safety and Efficacy of ZVS203e in the Treatment of Retinitis Pigmentosa Caused by RHO Gene Mutation
Recruiting now Phase 1 / Phase 2 Gene editing, Gene therapy
Chigenovo Co., Ltd. Gene: RHO. China
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A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Recruiting now Phase 2 Optogenetics
Kiora Pharmaceuticals, Inc.. Australia
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Safety and Efficacy of Stem Cell Small Extracellular Vesicles in Patients With Retinitis Pigmentosa
Recruiting now Phase 1 / Phase 2 Cell therapy
Mahidol University. Thailand
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A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Recruiting now Phase 1 / Phase 2 Drug / neuroprotection
Octant, Inc.. Gene: RHO. United States
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Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
Recruiting now Phase 1 / Phase 2 Gene therapy
Opus Genetics, Inc. Gene: LCA5. United States
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Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
Recruiting now Phase 1 / Phase 2 Gene therapy
Opus Genetics, Inc. Gene: RDH12. United States
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Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy
Recruiting now Phase 1 / Phase 2 Gene therapy
SparingVision. Belgium, France, Ireland
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A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa
Recruiting now Phase 1 / Phase 2 Gene therapy, Cell therapy
Sumitomo Pharma America, Inc.. United States
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Disulfiram for Treatment of Retinal Degeneration
Recruiting now Phase 1 / Phase 2 Drug / neuroprotection
University of Washington. United States
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Safety Evaluation Study of Pitavastatin PLGA Nano-particle for Retinitis Pigmentosa
Recruiting now Phase 1 Drug / neuroprotection
Kyushu University. Japan
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An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa
Recruiting now Phase 1 Drug / neuroprotection
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. China
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Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
Recruiting now Phase 1 Supplement / diet
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm). Australia, China, United States
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Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Exosomes for NAION
Recruiting now Phase 1 Cell therapy
The Foundation for Orthopaedics and Regenerative Medicine. Greece, United States
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The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration
Recruiting now Phase 1 Drug / neuroprotection
University of Rochester. United States
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Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Recruiting now Phase 1 Gene therapy
VeonGen Therapeutics GmbH. Gene: CNGA1. Germany
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Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
Recruiting now Early Phase 1 Optogenetics, Gene therapy
Augelux Therapeutics. Not gene-specific. China
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First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration
Recruiting now Early Phase 1 Gene therapy
Axovia Therapeutics. Gene: BBS1. United Kingdom
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Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
Recruiting now Early Phase 1 Gene therapy
Frontera Therapeutics. Gene: RPGR. China
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Safety and Efficacy Study of PUMCH-E111 Injection in Subjects With RLBP1 Related Inherited Retinal Dystrophy
Recruiting now Early Phase 1 Drug / neuroprotection
Peking Union Medical College Hospital. Gene: RLBP1. China
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Safety and Tolerability Study of Gene Editing Drug ZVS203e in Participants With Retinitis Pigmentosa
Recruiting now Early Phase 1 Gene editing, Gene therapy
Peking University Third Hospital. Gene: RHO. China
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A Study of LX107 Gene Therapy in AIPL1-IRD Patients
Recruiting now Early Phase 1 Gene therapy
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. Gene: AIPL1. China
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Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)
Recruiting now Observational Natural history / observational
Ascidian Therapeutics, Inc. United States
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Angiogenic Biomarkers in Juvenile Idiopathic Arthritis
Recruiting now Observational Natural history / observational
Assistance Publique - Hôpitaux de Paris. France
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High Resolution, High-speed Multimodal Ophthalmic Imaging
Recruiting now N/A Device
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
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Natural History Study of Usher Syndrome ( Light4Deaf )
Recruiting now Observational Natural history / observational
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
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Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7A
Recruiting now Observational Natural history / observational
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. Gene: MYO7A. France
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Implementation and Evaluation of a Post-Diagnostic Announcement Protocol at the CRMR RefeRet, Quinze-Vingts Hospital
Recruiting now N/A Other
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
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ARPKD Database Study
Recruiting now Observational Natural history / observational
Children's Hospital of Philadelphia. United States
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North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Recruiting now Observational Natural history / observational
Columbia University. Canada, United States
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Study of BEST1 Vitelliform Macular Dystrophy
Recruiting now Observational Natural history / observational
Columbia University. Gene: BEST1. France, Germany, United States
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Pilot Study of AuTNA I
Recruiting now N/A Gene therapy, Retinal implant
Eye & ENT Hospital of Fudan University. China
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Micropulsed Laser in Patients With Macular Oedema in Retinal Dystrophies
Recruiting now N/A Device
Fondazione G.B. Bietti, IRCCS. Italy
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Adaptive Optics Imaging of Outer Retinal Diseases
Recruiting now Observational Natural history / observational
Food and Drug Administration (FDA). United States
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Inherited Retinal Degenerative Disease Registry
Recruiting now Observational Natural history / observational
Foundation Fighting Blindness. United States
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Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Recruiting now Observational Natural history / observational
Hadassah Medical Organization. Israel
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EyeConic: Qualification for Cone-Optogenetics
Recruiting now Observational Natural history / observational
Institute of Molecular and Clinical Ophthalmology Basel. Not gene-specific. China, Germany, Hungary, Italy and 3 more
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Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
Recruiting now Observational Natural history / observational
Jaeb Center for Health Research. Australia, Belgium, Brazil, Canada and 10 more
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Global Registry and Natural History Study for Mitochondrial Disorders
Recruiting now Observational Natural history / observational
LMU Klinikum. Austria, Germany, Italy
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Stem Cell Ophthalmology Treatment Study II
Recruiting now N/A Cell therapy
MD Stem Cells. Austria, United Arab Emirates, United States
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Clinical Registry Investigating Bardet-Biedl Syndrome
Recruiting now Observational Natural history / observational
Marshfield Clinic Research Foundation. United States
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Cell Collection to Study Eye Diseases
Recruiting now Observational Natural history / observational
National Eye Institute (NEI). United States
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Rod and Cone Mediated Function in Retinal Disease
Recruiting now Observational Natural history / observational
National Eye Institute (NEI). United States
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A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Recruiting now Observational Natural history / observational
National Human Genome Research Institute (NHGRI). Gene: IMPDH1. United States
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Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance
Recruiting now Observational Natural history / observational
Okuvision GmbH. Germany
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Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
Recruiting now Observational Natural history / observational
Ray Therapeutics, Inc.. United States
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Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)
Recruiting now Observational Natural history / observational
Ray Therapeutics, Inc.. United States
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting now Observational Natural history / observational
Sanford Health. Gene: CRB1. United States
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Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration
Recruiting now N/A Retinal implant
Science Corporation. Australia
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Natural History Study of Patients With EYS-Associated RP
Recruiting now Observational Natural history / observational
Sensor Technology for Deafblind. Gene: EYS. Russia
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Evaluation of Objective Perimetry Using Chromatic Multifocal Pupillometer
Recruiting now Observational Natural history / observational
Sheba Medical Center. Israel
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Natural History Study of Advanced RCD
Recruiting now N/A Natural history / observational
SparingVision. Belgium, France, Ireland
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Monitoring and Early Detection of Treatable Inherited RETinal Disease in Children and Adolescents
Recruiting now Observational Natural history / observational
University College, London. Gene: RPE65, RPGR. Germany, United Kingdom, United States
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COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study
Recruiting now N/A Other
University Hospital, Strasbourg, France. France
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Retinal Imaging in Patients With Inherited Retinal Degenerations
Recruiting now Observational Natural history / observational
University of California, San Francisco. United States
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InsightRP2 Registry
Recruiting now Observational Natural history / observational
University of Göttingen. Gene: RP2. Germany
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Experimental and Clinical Studies of Retinal Stimulation
Recruiting now N/A Retinal implant
University of Michigan. United States
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High Resolution Retinal Imaging
Recruiting now Observational Natural history / observational
University of Pennsylvania. United States
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Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment
Recruiting now N/A Electrical stimulation
University of Waterloo. Canada
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Impact of Capsular Tension Ring on Intraocular Lens Position in Retinitis Pigmentosa Cataract Patients
Recruiting now N/A Other
Zhongshan Ophthalmic Center, Sun Yat-sen University. China
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GROWing Up With Rare GENEtic Syndromes
Recruiting now Observational Natural history / observational
dr. Laura C. G. de Graaff-Herder. Netherlands
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24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Not yet recruiting Phase 3 Drug / neuroprotection
Nacuity Pharmaceuticals, Inc.. Not gene-specific
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Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II
Not yet recruiting Phase 2 / Phase 3 Cell therapy
PT. Prodia Stem Cell Indonesia. Indonesia
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A Multicenter, Randomized, Double-Blind, Controlled Clinical Study of Minocycline for the Treatment of Retinitis Pigmentosa
Not yet recruiting Phase 3 Drug / neuroprotection
Zhongshan Ophthalmic Center, Sun Yat-sen University. China
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Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)
Not yet recruiting Phase 1 / Phase 2 Optogenetics, Gene therapy
Augelux Therapeutics. Not gene-specific
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Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Not yet recruiting Phase 2 RNA / antisense
Laboratoires Thea. Gene: USH2A
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The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis Pigmentosa
Not yet recruiting Phase 1 / Phase 2 Drug / neuroprotection
Sheba Medical Center. Israel
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Neoadjuvant RP2 + Nivolumab in Patients With Melanoma
Not yet recruiting Phase 2 Drug / neuroprotection
Yana Najjar. Gene: RP2. United States
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Phase I/II Open-Label Trial of UGX202 for Advanced Retinitis Pigmentosa
Not yet recruiting Phase 1 Drug / neuroprotection
Suzhou UgeneX Therapeutics Co., Ltd.
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UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
Not yet recruiting Early Phase 1 Gene therapy
Suzhou UgeneX Therapeutics Co., Ltd.. China
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Functional Assessments in Vision Impairment
Not yet recruiting Observational Natural history / observational
Center for Eye Research Australia. Australia
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A New Diagnostic Paradigm for Retinitis Pigmentosa Secondary to USH2A Pathogenic Variants
Not yet recruiting Observational Natural history / observational
IRCCS Ospedale San Raffaele. Gene: USH2A
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Inherited Retinal Diseases: Natural History and Genotype-Phenotype Correlations
Not yet recruiting Observational Natural history / observational
IRCCS San Raffaele. Gene: BEST1, PRPH2, RPE65, RPGR, USH2A. Italy
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Wide Field OCTA in Ocular Diseases
Not yet recruiting Observational Natural history / observational
IRCCS San Raffaele
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Retinal Investigation Using Optos OCT Device
Not yet recruiting N/A Device
Optos, PLC
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Long-term Safety of UC-MSC Transplantation in Patients With Retinitis Pigmentosa
Not yet recruiting Observational Natural history / observational
PT. Prodia Stem Cell Indonesia. Indonesia
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An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA
Not yet recruiting N/A Gene therapy
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. Gene: RPE65
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Non-invasive Ultrasound Retinal Stimulation for Vision Restoration
Not yet recruiting N/A Device
University of Southern California
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A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Enrolling by invitation Phase 2 Gene therapy
Beacon Therapeutics. Gene: RPGR. United States
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9-cis Beta-Carotene-Rich Extract of Dunaliella Alga in Retinitis Pigmentosa Patients
Enrolling by invitation Phase 1 / Phase 2 Supplement / diet, Drug / neuroprotection
Sheba Medical Center. Israel
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Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Enrolling by invitation Early Phase 1 RNA / antisense
University of Colorado, Denver. United States
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Longitudinal Study of a Bionic Eye
Enrolling by invitation Observational Natural history / observational
Center for Eye Research Australia. Australia
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Subthreshold Micropulse Laser Therapy (SML) in Retinitis Pigmentosa
Enrolling by invitation N/A Device
Marta P. Wiącek. Poland
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A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection
Enrolling by invitation Observational Natural history / observational
Nanoscope Therapeutics Inc.. Not gene-specific. India
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Prospective Exploratory Cohort Study on Ganglion Cell Degeneration in Retinitis Pigmentosa Patients
Enrolling by invitation Observational Natural history / observational
University Medical Center Goettingen. Not gene-specific. Germany
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A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
Underway, not enrolling Phase 2 / Phase 3 Gene therapy
Beacon Therapeutics. Gene: RPGR. Australia, Canada, United Kingdom, United States
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Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Underway, not enrolling Phase 3 Gene therapy
Innostellar Biotherapeutics Co.,Ltd. Gene: RPE65. China
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Oral N-acetylcysteine for Retinitis Pigmentosa
Underway, not enrolling Phase 3 Drug / neuroprotection
Johns Hopkins University. Not gene-specific. Austria, Canada, Germany, Netherlands and 3 more
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Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Underway, not enrolling Phase 3 Gene therapy
MeiraGTx Ocular UK Ltd. Gene: RPGR. Belgium, Canada, Denmark, France and 7 more
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A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa
Underway, not enrolling Phase 3 Gene therapy
MeiraGTx Ocular UK Ltd. Gene: RPGR. Japan
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A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
Underway, not enrolling Phase 3 Gene therapy
Ocugen. Not gene-specific. Canada, United States
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A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
Underway, not enrolling Phase 2 / Phase 3 RNA / antisense
ProQR Therapeutics. Gene: CEP290. Belgium, Brazil, Canada, France and 5 more
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Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Underway, not enrolling Phase 3 Gene therapy
Spark Therapeutics, Inc.. Gene: RPE65. United States
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4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
4D Molecular Therapeutics. Gene: RPGR. United States
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Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
Atsena Therapeutics Inc.. Gene: GUCY2D. United States
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Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
Beacon Therapeutics. Gene: RPGR. United States
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A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
Underway, not enrolling Phase 2 Gene therapy
Beacon Therapeutics. Gene: RPGR. United States
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A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
Underway, not enrolling Phase 2 Gene therapy
Beacon Therapeutics. Gene: RPGR. United States
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Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation
Underway, not enrolling Phase 1 / Phase 2 Cell therapy
Centre d'Etude des Cellules Souches. France
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Intravitreal Adalimumab in Inherited and Degenerative Retinal Diseases
Underway, not enrolling Phase 1 / Phase 2 Drug / neuroprotection
Centro de Pesquisa Rubens Siqueira. Brazil
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Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Underway, not enrolling Phase 1 / Phase 2 Optogenetics, Gene therapy
GenSight Biologics. Not gene-specific. France, United Kingdom, United States
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Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal Dystrophy
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
Innostellar Biotherapeutics Co.,Ltd. Gene: RPE65. China
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Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Underway, not enrolling Phase 2 RNA / antisense
Laboratoires Thea. Gene: USH2A. Belgium, Brazil, Canada, Denmark and 6 more
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A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
Underway, not enrolling Phase 2 Gene therapy
MeiraGTx Ocular UK Ltd. Gene: RPGR. United Kingdom, United States
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Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
Ocugen. Not gene-specific. United States
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A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001
Underway, not enrolling Phase 1 / Phase 2 RNA / antisense
PYC Therapeutics. Gene: PRPF31. United States
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A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
Underway, not enrolling Phase 1 / Phase 2 RNA / antisense
ProQR Therapeutics. Gene: RHO. United States
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Subtenon Autologous Platelet-Rich Plasma in Inherited and Degenerative Retinal Diseases
Underway, not enrolling Phase 1 / Phase 2 Drug / neuroprotection
Rubens Camargo Siqueira. Brazil
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PDE6A Gene Therapy for Retinitis Pigmentosa
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
STZ eyetrial. Gene: PDE6A. Germany
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A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
Underway, not enrolling Phase 2 Drug / neuroprotection
Sanofi. France, United States
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Promising ROd-cone DYstrophy Gene therapY
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
SparingVision. Gene: PDE6A, PDE6B, RHO. France, United States
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Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
Spark Therapeutics, Inc.. Gene: RPE65. United States
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Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)
Underway, not enrolling Phase 2 Cell therapy
jCyte, Inc. Not gene-specific. United States
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CNS10-NPC for the Treatment of RP
Underway, not enrolling Phase 1 Gene therapy, Cell therapy
Cedars-Sinai Medical Center. United States
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Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Underway, not enrolling Phase 1 Gene therapy
Innostellar Biotherapeutics Co.,Ltd. Gene: RPE65. China
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FIGHT-RP 1 Extension Study
Underway, not enrolling Phase 1 Drug / neuroprotection
Johns Hopkins University. Not gene-specific. United States
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Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis Pigmentosa
Underway, not enrolling Phase 1 Gene therapy, Cell therapy
Qi Zhou. China
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Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)
Underway, not enrolling Phase 1 Optogenetics, Gene therapy
Ray Therapeutics, Inc.. Not gene-specific. United States
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Clinical Trial of Autologous Intravitreal Bone-marrow CD34+ Stem Cells for Retinopathy
Underway, not enrolling Phase 1 Cell therapy
University of California, Davis. United States
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Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
Underway, not enrolling Early Phase 1 Gene therapy
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine. Gene: RPE65. China
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Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Underway, not enrolling Observational Natural history / observational
Genentech, Inc.. Gene: RPE65
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Psychotherapy Group for Parents of Children With LCA
Underway, not enrolling N/A Other
Instituto de Genética Ocular. Brazil
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Rate of Progression in USH2A-related Retinal Degeneration
Underway, not enrolling Observational Natural history / observational
Jaeb Center for Health Research. Gene: USH2A. Canada, France, Germany, Netherlands and 2 more
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Rate of Progression in EYS Related Retinal Degeneration
Underway, not enrolling Observational Natural history / observational
Jaeb Center for Health Research. Gene: EYS. Canada, Finland, France, Germany and 3 more
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Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F
Underway, not enrolling Observational Natural history / observational
Jaeb Center for Health Research. Gene: PCDH15. Canada, France, Germany, Israel and 4 more
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Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
Underway, not enrolling Observational Natural history / observational
Nanoscope Therapeutics Inc.. Not gene-specific. Puerto Rico, United States
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Electro-acupuncture and Transcorneal Electrical Stimulation (TES) for Retinitis Pigmentosa
Underway, not enrolling N/A Electrical stimulation
Nova Southeastern University. United States
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Natural History Study of Retinitis Pigmentosa Type 11
Underway, not enrolling Observational Natural history / observational
Oslo University Hospital. Gene: PRPF31. Norway
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Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
Underway, not enrolling Observational Natural history / observational
PYC Therapeutics. Gene: PRPF31. Australia, United States
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Early Check: Expanded Screening in Newborns
Underway, not enrolling Observational Natural history / observational
RTI International. United States
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Early Genetic Identification of Obesity
Underway, not enrolling Observational Natural history / observational
Rolfs Consulting und Verwaltungs-GmbH (RCV). Germany
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Prospective Natural History Study of Retinitis Pigmentosa
Underway, not enrolling N/A Natural history / observational
SparingVision. Gene: PDE6A, PDE6B, RHO. France, United States
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A Prospective, Observational Study in Adults With Retinitis Pigmentosa (RP)
Underway, not enrolling Observational Natural history / observational
Sumitomo Pharma America, Inc.. United States
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Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa
Underway, not enrolling Observational Natural history / observational
University Hospital Tuebingen. Gene: PDE6A, PDE6B, RHO. Germany
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The Role of Capsular Tension Ring (CTR) in Anterior Capsular Contraction
Completed Phase 4 Other
Aurolab. India
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Management of Retinitis Pigmentosa by Mesenchymal Stem Cells by Wharton's Jelly Derived Mesenchymal Stem Cells
Completed Phase 3 Cell therapy
Ankara Universitesi Teknokent. Turkey (Türkiye)
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Management of Retinitis Pigmentosa Via Combination of Wharton's Jelly-derived Mesenchymal Stem Cells and Magnovision
Completed Phase 3 Cell therapy
Ankara Universitesi Teknokent. Turkey (Türkiye)
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Randomized Trial of DHA for Retinitis Pigmentosa Patients Receiving Vitamin A
Completed Phase 3 Supplement / diet
Carol Weigel DiFranco. United States
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CB-PRP in Retinitis Pigmentosa and Dry Age-related Macular Degeneration
Completed Phase 3 Gene therapy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS. Italy
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Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
Terminated Phase 2 / Phase 3 RNA / antisense
Laboratoires Thea. Gene: USH2A. Germany, Netherlands, United Kingdom, United States
-
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
Terminated Phase 2 / Phase 3 RNA / antisense
Laboratoires Thea. Gene: USH2A. United Kingdom, United States
-
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Terminated Phase 2 / Phase 3 Drug / neuroprotection
Liminal BioSciences Ltd.. United Kingdom
-
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Completed Phase 3 Gene therapy
MeiraGTx Ocular UK Ltd. Gene: RPGR. Belgium, Canada, Denmark, France and 7 more
-
Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis Pigmentosa
Completed Phase 3 Supplement / diet
National Eye Institute (NEI)
-
Clinical Trial of Lutein for Patients With Retinitis Pigmentosa Receiving Vitamin A
Completed Phase 3 Supplement / diet
National Eye Institute (NEI). United States
-
Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
Completed Phase 3 Gene therapy
NightstaRx Ltd, a Biogen Company. Gene: CHM, RPGR. Brazil, Canada, Denmark, Finland and 5 more
-
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
Status unknown Phase 2 / Phase 3 RNA / antisense
ProQR Therapeutics. Gene: CEP290. Belgium, Brazil, Canada, Germany and 3 more
-
Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
Terminated Phase 3 Drug / neuroprotection
R-Tech Ueno, Ltd.. Japan
-
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
Completed Phase 3 Drug / neuroprotection
Rhythm Pharmaceuticals, Inc.. Canada, France, Puerto Rico, Spain and 2 more
-
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
Completed Phase 3 Drug / neuroprotection
Rhythm Pharmaceuticals, Inc.. Australia, Spain, United Kingdom, United States
-
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
Completed Phase 3 Drug / neuroprotection
Rhythm Pharmaceuticals, Inc.. Canada, Germany, Netherlands, Puerto Rico and 2 more
-
The Effect of Stem Cells and Stem Cell Exosomes on Visual Functions in Patients With Retinitis Pigmentosa
Status unknown Phase 2 / Phase 3 Cell therapy
TC Erciyes University. Turkey (Türkiye)
-
AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Optogenetics, Gene therapy
AbbVie. Not gene-specific. United States
-
Spheroidal Mesenchymal Stem Cells in Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Cell therapy
Acibadem University. Turkey (Türkiye)
-
A Study in Subjects With Retinitis Pigmentosa
Completed Phase 2 Drug / neuroprotection
Aldeyra Therapeutics, Inc.. United States
-
An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Drug / neuroprotection
Allergan. France, Germany, Portugal, United States
-
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Completed Phase 1 / Phase 2 Gene therapy
Beacon Therapeutics. Gene: RPE65. United States
-
Studying a Potential Protective Effect of L-Dopa on Retinitis Pigmentosa
Status unknown Phase 2 Drug / neuroprotection
Beirut Eye Specialist Hospital. Lebanon
-
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
Completed Phase 1 / Phase 2 Gene therapy
Biogen. Gene: RPGR. United Kingdom, United States
-
Clinical Study to Evaluate Safety and Efficacy of BMMNC in Retinitis Pigmentosa
Status unknown Phase 1 / Phase 2 Cell therapy
Chaitanya Hospital, Pune. India
-
Sildenafil for Treatment of Choroidal Ischemia
Completed Phase 2 Drug / neuroprotection
Columbia University. United States
-
A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)
Completed Phase 1 / Phase 2 Drug / neuroprotection
Dompé Farmaceutici S.p.A. Italy
-
Single Ascending Dose Study in Participants With LCA10
Status unknown Phase 1 / Phase 2 Gene editing, Gene therapy
Editas Medicine, Inc.. Gene: CEP290. United States
-
Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa
Terminated Phase 1 / Phase 2 Drug / neuroprotection
Endogena Therapeutics, Inc. United States
-
Trial of Oral Valproic Acid for Retinitis Pigmentosa
Completed Phase 2 Drug / neuroprotection
Foundation Fighting Blindness. United States
-
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
Status unknown Phase 1 / Phase 2 Gene therapy
Frontera Therapeutics. Gene: RPGR. China
-
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Status unknown Phase 1 / Phase 2 Gene therapy
HuidaGene Therapeutics Co., Ltd.. Gene: RPE65. China, United States
-
Investigation of Therapeutic Efficacy and Safety of UMSCs for the Management of Retinitis Pigmentosa (RP)
Status unknown Phase 2 Gene therapy, Cell therapy
Jinnah Burn and Reconstructive Surgery Centre, Lahore. Pakistan
-
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
Completed Phase 1 / Phase 2 Optogenetics
Kiora Pharmaceuticals, Inc.. Australia
-
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
Completed Phase 1 / Phase 2 RNA / antisense
Laboratoires Thea. Gene: CEP290. Belgium, United States
-
Extension Study to Study PQ-110-001 (NCT03140969)
Terminated Phase 1 / Phase 2 RNA / antisense
Laboratoires Thea. Gene: CEP290. Belgium, United States
-
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
Terminated Phase 2 RNA / antisense
Laboratoires Thea. Gene: USH2A. Canada, France, United States
-
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Completed Phase 1 / Phase 2 Gene therapy
MeiraGTx UK II Ltd. Gene: RPE65. United Kingdom, United States
-
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
Completed Phase 1 / Phase 2 Gene therapy
MeiraGTx UK II Ltd. Gene: RPGR. United Kingdom, United States
-
Aflibercept for Macular Oedema With Underlying Retinitis Pigmentosa (AMOUR) Study
Completed Phase 2 Drug / neuroprotection
Moorfields Eye Hospital NHS Foundation Trust. United Kingdom
-
Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
Completed Phase 1 / Phase 2 Drug / neuroprotection
Nacuity Pharmaceuticals, Inc.. Not gene-specific. Australia
-
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Optogenetics, Gene therapy
Nanoscope Therapeutics Inc.. Not gene-specific. India
-
Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Completed Phase 2 Optogenetics, Gene therapy
Nanoscope Therapeutics Inc.. Not gene-specific. Puerto Rico, United States
-
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Completed Phase 1 / Phase 2 Gene therapy
Nantes University Hospital. Gene: RPE65. France
-
Effects of Lutein in Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Supplement / diet, Drug / neuroprotection
National Center for Complementary and Integrative Health (NCCIH). United States
-
Study of Oral Minocycline in Treating Bilateral Cystoid Macular Edema Associated With Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Drug / neuroprotection
National Eye Institute (NEI). United States
-
Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)
Terminated Phase 1 / Phase 2 Drug / neuroprotection
National Eye Institute (NEI). Gene: NR2E3. United States
-
A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
Completed Phase 2 Drug / neuroprotection
Neurotech Pharmaceuticals. United States
-
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
Completed Phase 2 Drug / neuroprotection
Neurotech Pharmaceuticals. United States
-
Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
Completed Phase 2 Cell therapy
Neurotech Pharmaceuticals. United States
-
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Gene therapy
Novartis Pharmaceuticals. Gene: RLBP1. Sweden
-
Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
Completed Phase 2 Drug / neuroprotection
Ospedale San Raffaele. Italy
-
Safety Issues of Peribulbar Injection of UC-MSC in Patients With Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Cell therapy
PT. Prodia Stem Cell Indonesia. Indonesia
-
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene
Completed Phase 1 / Phase 2 RNA / antisense
ProQR Therapeutics. Gene: USH2A. Canada, France, United States
-
Safety Study in Retinal Transplantation for Retinitis Pigmentosa.
Completed Phase 2 Device
Radtke, Norman D., M.D.. United States
-
Intravitreal Dexamethasone Implant in Retinitis Pigmentosa-related Macular Edema- a Retrospective Study
Status unknown Phase 2 Drug / neuroprotection
Rafic Hariri University Hospital
-
Safety and Tolerability of hRPC in Retinitis Pigmentosa
Status unknown Phase 1 / Phase 2 Gene therapy, Cell therapy, Retinal implant
ReNeuron Limited. Spain, United Kingdom, United States
-
DHA and X-Linked Retinitis Pigmentosa
Completed Phase 2 Supplement / diet
Retina Foundation of the Southwest. United States
-
Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
Terminated Phase 1 / Phase 2 Gene therapy
Sanofi. France, United States
-
Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa
Completed Phase 2 Drug / neuroprotection
Seoul National University Hospital. South Korea
-
Effects of Treatment With N- Acetylcysteine on Visual Outcomes in Patients With Retinitis Pigmentosa
Status unknown Phase 2 Drug / neuroprotection
Shahid Beheshti University of Medical Sciences. Not gene-specific. Iran
-
The Effect of Oral Administration of 9-cis β Carotene Rich Powder of the Alga Dunaliella Bardawil
Status unknown Phase 1 / Phase 2 Supplement / diet, Drug / neuroprotection
Sheba Medical Center. Israel
-
Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
Status unknown Phase 1 / Phase 2 Cell therapy
Stem Cells Arabia. Jordan
-
Minocycline Treatment in Retinitis Pigmentosa
Completed Phase 2 Drug / neuroprotection
Sun Yat-sen University. China
-
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Completed Phase 1 / Phase 2 Gene therapy
University College, London. Gene: RPE65. United Kingdom
-
Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement
Withdrawn Phase 2 Drug / neuroprotection
University Hospital Tuebingen
-
Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)
Status unknown Phase 2 Supplement / diet, Drug / neuroprotection
University of Colorado, Denver. United States
-
Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)
Terminated Phase 1 / Phase 2 Drug / neuroprotection
University of Michigan. Gene: RHO. United States
-
Gene Therapy for Blindness Caused by Choroideremia
Completed Phase 1 / Phase 2 Gene therapy
University of Oxford. United Kingdom
-
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
Completed Phase 2 Cell therapy
University of Sao Paulo. Brazil
-
Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene
Terminated Phase 1 / Phase 2 Gene therapy
eyeDNA Therapeutics. Gene: PDE6B. France
-
Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa
Completed Phase 1 / Phase 2 Cell therapy
jCyte, Inc. Not gene-specific. United States
-
Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa
Completed Phase 2 Cell therapy
jCyte, Inc. Not gene-specific. United States
-
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
Completed Phase 2 Cell therapy
jCyte, Inc. Not gene-specific. United States
-
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
Completed Phase 1 Drug / neuroprotection
Abliva AB. United Kingdom
-
A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis Pigmentosa
Terminated Phase 1 Gene therapy
Centocor, Inc.. United States
-
BF844 Safety and Pharmacokinetic Study in Healthy Volunteers
Status unknown Phase 1 Drug / neuroprotection
EyeXCel Pty. Ltd.. Australia
-
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
Completed Phase 1 Gene therapy
Hadassah Medical Organization. Gene: RPE65. Israel
-
Acupuncture for the Treatment of Vision Loss Due to Retinitis Pigmentosa
Completed Phase 1 Other
Johns Hopkins University. United States
-
The FIGHT-RP1 Study
Completed Phase 1 Drug / neuroprotection
Johns Hopkins University. Not gene-specific. United States
-
Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)
Completed Phase 1 Gene therapy
King Khaled Eye Specialist Hospital. Gene: MERTK. Saudi Arabia
-
Intravitreal Injection of MSCs in Retinitis Pigmentosa
Completed Phase 1 Cell therapy
Mahidol University. Thailand
-
Evaluation of Safety of Ciliary Neurotrophic Factor Implants in the Eye
Completed Phase 1 Drug / neuroprotection
National Eye Institute (NEI). United States
-
Investigating the Effect of Vitamin A Supplementation on Retinitis Pigmentosa
Completed Phase 1 Supplement / diet, Drug / neuroprotection
National Eye Institute (NEI). United States
-
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
Completed Phase 1 Drug / neuroprotection
PYC Therapeutics. Gene: PRPF31. United States
-
Stem Cells Therapy in Degenerative Diseases of the Retina
Status unknown Phase 1 Cell therapy
Pomeranian Medical University Szczecin. Poland
-
Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations
Completed Phase 1 Drug / neuroprotection
QLT Inc.. Gene: RPE65. Canada, Germany, Netherlands, United Kingdom and 1 more
-
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
Completed Phase 1 Drug / neuroprotection
QLT Inc.. Gene: RPE65. Canada, Germany, Netherlands, United Kingdom and 1 more
-
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
Completed Phase 1 Drug / neuroprotection
QLT Inc.. Gene: RPE65. Canada, Ireland
-
Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
Completed Phase 1 Cell therapy
Red de Terapia Celular. Spain
-
Safety Study in Subjects With Leber Congenital Amaurosis
Completed Phase 1 Gene therapy
Spark Therapeutics, Inc.. Gene: RPE65. United States
-
Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa
Completed Phase 1 Cell therapy
University of California, Davis. United States
-
Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
Completed Phase 1 Gene therapy
University of Pennsylvania. Gene: RPE65. United States
-
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
Completed Phase 1 Cell therapy
University of Sao Paulo. Brazil
-
Treatment of RP and LCA by Primary RPE Transplantation
Status unknown Early Phase 1 Gene therapy
Eyecure Therapeutics Inc.. China
-
The Effects of Cannabis on Visual Functions in Healthy and Retinitis Pigmentosa Patients
Status unknown Early Phase 1 Drug / neuroprotection
Hadassah Medical Organization. Israel
-
Clinical Trials of Continuous Oxygen Therapy Combined With Blue Light Deprivation in the Treatment of Retinitis Pigmentosa
Status unknown Early Phase 1 Supplement / diet, Drug / neuroprotection
Sun Yat-sen University. China
-
Intravitreal Injection of SeeQ CdSe 655 Alt Nanoparticles for Patients With Degenerative Retinal Diseases
Completed N/A Drug / neuroprotection
2C Tech Corp. Mexico
-
Intravitreal Quantum Dots (QD) for Advanced Retinitis Pigmentosa (RP) (QUANTUM)
Withdrawn N/A Drug / neuroprotection
2C Tech Corp
-
A New Treatment of Retinitis Pigmentosa
Status unknown N/A Other
Aier School of Ophthalmology, Central South University
-
Bardet Beidle Syndrome in a Syrian Adolescent : a Rare Case Report
Status unknown Observational Natural history / observational
Al Baath University
-
Management of Cystoid Macular Edema Secondary to Retinitis Pigmentosa Via Subliminal Micropulse Yellow Laser
Completed N/A Device
Ankara Universitesi Teknokent. Turkey (Türkiye)
-
Effects of Subtenon-injected Autologous Platelet-rich Plasma on Visual Functions in Eyes With Retinitis Pigmentosa
Completed N/A Drug / neuroprotection
Ankara Universitesi Teknokent. Turkey (Türkiye)
-
Management of Retinitis Pigmentosa Via Electromagnetic Stimulation and Platelet Rich Plasma
Completed N/A Drug / neuroprotection
Ankara Universitesi Teknokent
-
Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
Completed Observational Natural history / observational
Assistance Publique - Hôpitaux de Paris. France
-
Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM)
Completed N/A Other
Assistance Publique - Hôpitaux de Paris. France
-
Development of Visual Function Evaluation Method
Completed Observational Natural history / observational
Astellas Pharma Inc. Japan
-
Development of Visual Function Evaluation Method (2)
Completed Observational Natural history / observational
Astellas Pharma Inc. Japan
-
Slowing the Degenerative Process, Long Lasting Effect of Hyperbaric Oxygen Therapy in Retinitis Pigmentosa
Completed N/A Other
Azienda Policlinico Umberto I. Italy
-
Structural and Functional Characterization of Rare Ocular Diseases
Status unknown Observational Natural history / observational
Barcelona Macula Foundation. Spain
-
Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Completed Observational Natural history / observational
Beacon Therapeutics. Gene: RPGR. United States
-
Pilot Study of a Suprachoroidal Retinal Prosthesis
Completed N/A Retinal implant
Center for Eye Research Australia. Australia
-
Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in Europe
Status unknown Observational Natural history / observational
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France, Germany, Netherlands, Portugal
-
High Resolution Retina Imaging
Status unknown Observational Natural history / observational
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
-
Impact of Visual Field Restriction on Visual Exploration
Status unknown Observational Natural history / observational
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
-
Mobility Protocol Adapted for Advanced Visually Impaired Subjects
Status unknown N/A Other
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
-
Validation of Standardized Test Protocols to Assess the Impact of Visual Pathologies in Daily Life Activities
Status unknown N/A Gene therapy, Retinal implant
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
-
Bimodal and Coaxial High Resolution Ophtalmic Imaging
Status unknown N/A Other
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
-
Pupil Dynamics and Color Vision for the Detection of Eye Diseases
Completed N/A Other
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts. France
-
AUgmented REality for the Visually Impaired - Part 1
Completed N/A Device
Centre Hospitalier Universitaire de Nīmes. France
-
Evoked Retinal Response
Terminated N/A Other
Columbia University. United States
-
CNGB1 and Allied Disorders
Suspended Observational Natural history / observational
Columbia University. Gene: CNGB1. France, Germany, United Kingdom, United States
-
SS-HH-OCT as a Novel Diagnostic Modality for Early-Onset Retinal Dystrophies (EORDs)
Completed N/A Device
Duke University. United States
-
Natural History Study of CEP290-Related Retinal Degeneration
Completed Observational Natural history / observational
Editas Medicine, Inc.. Gene: CEP290. France, Germany, Netherlands, United States
-
Exercise and RP (AVAMC and Emory)
Completed N/A Other
Emory University. United States
-
Biomarkers in Retinitis Pigmentosa
Status unknown Observational Natural history / observational
Fondazione Policlinico Universitario Agostino Gemelli IRCCS. Italy
-
Natural History Study in Subjects With Usher Syndrome
Completed Observational Natural history / observational
Fondazione Telethon. Italy, Netherlands, Spain
-
Study on the Effects of Mutations Under Inherited Retinal Disease in Korean
Completed Observational Natural history / observational
Gangnam Severance Hospital. South Korea
-
Identifying Genes and Mutations Underlying Retinitis Pigmentosa and Allied Diseases
Status unknown Observational Natural history / observational
Hillel Yaffe Medical Center
-
CArotenoid in hypoChOlesterolemia
Completed Observational Natural history / observational
Hospices Civils de Lyon. France
-
IRIS PILOT - Extended Pilot Study With a Retinal Implant System
Status unknown N/A Retinal implant, Electrical stimulation
Intelligent Medical Implants GmbH
-
Mechanisms of Inherited Retinal Dystrophies Using Whole Genome Sequencing and in Vitro and in Vivo Models
Completed Observational Natural history / observational
Istituto Superiore di Sanità. Italy
-
An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
Completed Observational Natural history / observational
Janssen Pharmaceutical K.K.. Gene: RPGR. Japan
-
A Study of Pentosan Polysulfate Sodium and the Development of Pigmentary Maculopathy and Pigmentary Retinopathy
Completed Observational Natural history / observational
Janssen Research & Development, LLC. United States
-
Stress and Vision Fluctuations in Retinitis Pigmentosa
Completed Observational Natural history / observational
Johns Hopkins University. United States
-
Biomarkers In Retinitis Pigmentosa (BIRP)
Completed Observational Natural history / observational
Johns Hopkins University. United States
-
Environmental Localization Mapping and Guidance for Visual Prosthesis Users
Completed N/A Retinal implant
Johns Hopkins University. United States
-
A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
Terminated Observational Natural history / observational
Johns Hopkins University. United States
-
Stem Cell Ophthalmology Treatment Study
Status unknown N/A Gene therapy, Cell therapy
MD Stem Cells. United States
-
Collision Warning Device for Blind and Visually Impaired
Completed N/A Device
Massachusetts Eye and Ear Infirmary. United States
-
Argus II/ORCAM Device Study
Completed N/A Retinal implant
Mayo Clinic. United States
-
Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine
Completed Observational Natural history / observational
Mayo Clinic. United States
-
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Completed Observational Natural history / observational
MeiraGTx Ocular UK Ltd. Gene: RPGR. United Kingdom, United States
-
Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
Completed Observational Natural history / observational
MeiraGTx UK II Ltd. Gene: RPE65. United Kingdom, United States
-
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Completed Observational Natural history / observational
MeiraGTx UK II Ltd. Gene: RPE65. United Kingdom, United States
-
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
Completed Observational Natural history / observational
MeiraGTx UK II Ltd. Gene: RPGR. Canada, United Kingdom, United States
-
Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.
Status unknown Observational Natural history / observational
MejoraVisionMD. Mexico
-
Object Finder for a Retinal Prosthesis
Completed N/A Retinal implant
Minnesota HealthSolutions. United States
-
Study of a Suprachoroidal Retinal Prosthesis
Completed N/A Retinal implant
Mobius Medical Pty Ltd.. Australia
-
NR600 System Retinal Prosthesis for Patients With Retinal Degenerative Diseases
Terminated N/A Retinal implant
Nano Retina. Belgium, Israel, Italy
-
Observational Study to Assess the Reliability and Validity of the MLYMT and MLSDT
Completed Observational Natural history / observational
Nanoscope Therapeutics Inc.. Puerto Rico, United States
-
Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome
Terminated Observational Natural history / observational
National Center for Research Resources (NCRR). United States
-
A Genetic Analysis of Usher Syndrome in Ashkenazi Jews
Completed Observational Natural history / observational
National Center for Research Resources (NCRR). United States
-
Gyrate Atrophy of the Choroid and Retina
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Study of Usher Syndromes, Type 1 and Type 2
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Natural History and Genetic Studies of Usher Syndrome
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Molecular Genetics of Retinal Degenerations
Completed Observational Natural history / observational
National Eye Institute (NEI). China, India, Pakistan, United States
-
Repository for Inherited Eye Diseases
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Potential Research Participants for Future Studies of Inherited Eye Diseases
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Color Vision as a Measure for Inherited Retinal Diseases
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures
Completed Observational Natural history / observational
National Eye Institute (NEI). United States
-
Vitamin Replacement in Abetalipoproteinemia
Completed Observational Natural history / observational
National Heart, Lung, and Blood Institute (NHLBI). United States
-
Genetics and Clinical Characteristics of Bardet-Biedl Syndrome
Terminated Observational Natural history / observational
National Human Genome Research Institute (NHGRI). United States
-
Genetic Analyses of Nonsyndromic and Syndromic Deafness in Pakistan
Completed Observational Natural history / observational
National Institute on Deafness and Other Communication Disorders (NIDCD). Pakistan
-
Study of the Correlation Between Macular Thickness Analyzed by Optical Coherence Tomography and the Visual Functions in Retinitis Pigmemtosa
Completed Observational Natural history / observational
National Taiwan University Hospital. Taiwan
-
Natural History of the Progression of X-Linked Retinitis Pigmentosa
Completed Observational Natural history / observational
NightstaRx Ltd, a Biogen Company. Gene: RPGR. Canada, Finland, France, Germany and 3 more
-
Expanded Access Program for RP in Adults
Expanded access ended N/A Gene therapy
Ocugen. Not gene-specific. United States
-
Auditory Neural Function in Implanted Patients With Usher Syndrome
Completed N/A Other
Ohio State University. United States
-
Transcorneal Electrical Stimulation Therapy for Retinal Disease
Completed N/A Electrical stimulation
Okuvision GmbH
-
Transcorneal Electrical Stimulation - Multicenter Safety Study
Completed N/A Electrical stimulation
Okuvision GmbH. Denmark, Germany, Norway
-
Transcorneal Electrostimulation for Therapy of Retinitis Pigmentosa
Completed N/A Electrical stimulation
Okuvision GmbH. Germany
-
Retrospective NIS to Evaluate the Patient Benefit of TES
Completed Observational Natural history / observational
Okuvision GmbH. Germany, Switzerland
-
Characterizing the Genotype and Phenotype in Adults With Bardet-Biedl Syndrome
Completed Observational Natural history / observational
Oslo University Hospital. Norway
-
Characterization and Functionality of Calcium Channels Cav1.4 of Th17 Lymphocytes in Human With Psoriasis
Completed N/A Drug / neuroprotection
Pierre Fabre Dermo Cosmetique. France
-
In Depth Observational Clinical Trial Of Retinitis Pigmentosa Patients
Status unknown Observational Natural history / observational
Power Life Sciences Inc.
-
Sema 4A as a Marker for Inflammatory Disease in Multiple Sclerosis
Withdrawn Observational Natural history / observational
Providence Health & Services. United States
-
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
Completed Observational Natural history / observational
QLT Inc.. Gene: RPE65. Canada, Denmark, Germany, Netherlands and 3 more
-
Visual Performance Measures in a Virtual Reality Environment for Assessing Clinical Trial Outcomes in Those With Severely Reduced Vision
Completed Observational Natural history / observational
Queen's University, Belfast. United Kingdom
-
Characterizing Rate of Progression in USHer Syndrome (CRUSH) Study
Status unknown Observational Natural history / observational
Radboud University Medical Center. Gene: USH2A. Netherlands
-
Study of Docosahexaenoic Acid (DHA) Supplementation in Patients With X-Linked Retinitis Pigmentosa
Completed N/A Supplement / diet
Retina Foundation of the Southwest
-
Rod Sensitivity in Age-related Macular Degeneration (AMD) and Retinitis Pigmentosa (RP)
Completed Observational Natural history / observational
Retina Foundation of the Southwest. United States
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Retina Implant Pilot Trial to Evaluate Safety & Efficacy in Blind Patients Having Degenerated Photo-receptors
Completed N/A Gene therapy, Retinal implant
Retina Implant AG. Germany
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Safety and Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients
Completed N/A Gene therapy, Retinal implant
Retina Implant AG. Germany, Hungary, United Kingdom
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Safety & Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients
Completed N/A Gene therapy, Retinal implant
Retina Implant AG. Hong Kong
-
PMCF Study for Partial Vision Restoration Using the Subretinal Implant RETINA IMPLANT Alpha AMS
Withdrawn Observational Natural history / observational
Retina Implant AG. Germany
-
Impact on Daily Life of Patients Using the Subretinal Implant RETINA IMPLANT Alpha AMS
Terminated N/A Gene therapy, Retinal implant
Retina Implant AG. France
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Microcurrent Stimulation to Treat Macular Degeneration
Completed Observational Natural history / observational
Retina Institute of Hawaii. United States
-
An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)
Approved for marketing N/A Drug / neuroprotection
Rhythm Pharmaceuticals, Inc.
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Clinical Characterization on PDE6A-related Retinitis Pigmentosa in Preparation to a Gene Therapy Trial
Completed Observational Natural history / observational
STZ eyetrial. Gene: PDE6A. Germany
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Restoring Vision With the Intelligent Retinal Implant System (IRIS V1)in Patients With Retinal Dystrophy
Completed N/A Retinal implant
Science Corporation. Austria, France, Germany
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Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy
Completed N/A Retinal implant
Science Corporation. Austria, France, Germany, Spain and 1 more
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Feasibility Study of a Chronic Retinal Stimulator in Retinitis Pigmentosa
Completed N/A Retinal implant, Electrical stimulation
Second Sight Medical Products. United States
-
Argus® II Retinal Stimulation System Feasibility Protocol
Completed N/A Retinal implant
Second Sight Medical Products. France, Mexico, Switzerland, United Kingdom and 1 more
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Argus® II Retinal Prosthesis System Post-Market Surveillance Study
Terminated Observational Natural history / observational
Second Sight Medical Products. Germany, Italy
-
New Enrollment Post-Approval Study of the Argus® II Retinal Prosthesis System
Terminated Observational Natural history / observational
Second Sight Medical Products. United States
-
Post-Market Study of the Argus® II Retinal Prosthesis System - France
Completed Observational Natural history / observational
Second Sight Medical Products. France
-
Argus II Retinal Prosthesis System - Better Vision RP Study
Withdrawn N/A Retinal implant
Second Sight Medical Products. Germany
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Autoimmunity in Retinitis Pigmentosa
Status unknown N/A Drug / neuroprotection
Semmelweis University. Hungary
-
Clinical and Genetic Testing of Patients With Usher Syndrome
Completed Observational Natural history / observational
Sensor Technology for Deafblind. Russia
-
Prospective Open Clinical and Genetic Study of Patients With Retinitis Pigmentosa
Completed Observational Natural history / observational
Sensor Technology for Deafblind. Russia
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Establishment of the National Registry for Inherited Retinal Dystrophy in Iran
Status unknown Observational Natural history / observational
Shahid Beheshti University of Medical Sciences. Iran
-
Evaluation of Objective Perimetry Using Pupillometer
Completed Observational Natural history / observational
Sheba Medical Center. Israel
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The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions im Patients With Retinitis Pigmentosa
Completed N/A Supplement / diet, Drug / neuroprotection
Sheba Medical Center. Israel
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Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)
Terminated Observational Natural history / observational
Shire. United States
-
Retrospective Natural History Study of Retinitis Pigmentosa
Status unknown Observational Natural history / observational
SparingVision. Gene: PDE6A, PDE6B, RHO. France
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A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Completed Observational Natural history / observational
Spark Therapeutics, Inc.. Gene: RPE65. United States
-
Computer-Assisted Rehabilitation Environment Training After Argus Retinal Prosthesis
Completed N/A Retinal implant
The Cleveland Clinic. United States
-
Cone Rescue in Retinitis Pigmentosa by the Treatment of Lycium Barbarum
Completed N/A Other
The Hong Kong Polytechnic University. Hong Kong
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The Effect of GLP1 Agonists on Weight Loss in BBS Cohort in the UK
Completed Observational Natural history / observational
The Queen Elizabeth Hospital. United Kingdom
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Effectiveness of a Cognitive-behavioral Program of Coping With Psychological Stress in People With Retinitis Pigmentosa
Completed N/A Other
Universidad de Granada. Spain
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Coloured-light in Retinitis Pigmentosa.
Status unknown N/A Device
University College, London. United Kingdom
-
Observational Study of the Argus® II Retinal Prosthesis System
Status unknown Observational Natural history / observational
University Health Network, Toronto. Canada
-
Clinical and Genetic Findings in Patients With PRPF31-associated Retinitis Pigmentosa
Completed Observational Natural history / observational
University Hospital Tuebingen. Gene: PRPF31. Germany
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ABCA4-associated Disease in Childhood and Adolescence - a Phenotype Study
Completed Observational Natural history / observational
University Hospital Tuebingen. Germany
-
Study of New Mutations in Cone Disorders
Terminated Observational Natural history / observational
University Hospital, Lille. France
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Evaluation of the Visual Field on the Ground
Withdrawn N/A Supplement / diet
University Hospital, Limoges
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Autosomal Dominant Retinitis Pigmentosa: Prevalence of Known Genes Identification of New Loci / Genes
Completed N/A Other
University Hospital, Montpellier. France
-
Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults
Completed Observational Natural history / observational
University Hospital, Strasbourg, France. Gene: BBS1. France
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Clinical Implication of Retinitis Pigmentosa Molecular Diagnostic Using High Throughput Sequencing.
Status unknown Observational Natural history / observational
University Hospital, Strasbourg, France. France
-
Clinical Study of a Single Ciliopathy: Alström Syndrome
Terminated Observational Natural history / observational
University Hospital, Strasbourg, France. France
-
Self-confidence Study in Patients With Argus II Artificial Retina
Status unknown Observational Natural history / observational
University Hospital, Strasbourg, France. France
-
The Perceptual Experience of Argus II Users
Completed N/A Retinal implant
University of California, Santa Barbara. United States
-
Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene
Completed Observational Natural history / observational
University of Campania Luigi Vanvitelli. Gene: RPE65. Italy
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Advancing Understanding of Transportation Options
Completed N/A Other
University of Colorado, Denver. United States
-
Visual Activity Evoked by Infrared in Humans After Dark Adaptation
Completed N/A Other
University of New Mexico. United States
-
TES for the Treatment of RP
Completed N/A Electrical stimulation
University of Oxford. United Kingdom
-
Visual and Functional Assessment in Low Vision Patients
Completed Observational Natural history / observational
University of Oxford. United Kingdom
-
Safety and Efficacy of the Alpha AMS Subretinal Implant
Completed N/A Gene therapy, Retinal implant
University of Oxford. United Kingdom
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Computer Aided Diagnosis of Multiple Eye Fundus Diseases From Color Fundus Photograph
Completed Observational Natural history / observational
Visionary Intelligence Ltd.. China
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Transcorneal Electrical Stimulation for the Treatment of Retinitis Pigmentosa - a Safety and Efficacy Multicenter Study
Withdrawn N/A Electrical stimulation
Wills Eye. United States
-
RETINA IMPLANT Alpha AMS in Blind Patients With Retinitis Pigmentosa
Withdrawn N/A Gene therapy, Retinal implant
Wills Eye. United States
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Evaluation of New Head-mounted Visual Aids Among Patients With Low Vision
Status unknown N/A Device
Zhongshan Ophthalmic Center, Sun Yat-sen University. China