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Conditions: Usher Syndrome, Type 2A, Retinitis Pigmentosa 39
Interventions: Not listed
Lead sponsor: Jaeb Center for Health Research; Other
Eligibility: 8 Years and older
Enrollment: 127 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2029
U.S. locations: 9
States / cities: San Francisco, California • Gainesville, Florida • Baltimore, Maryland + 6 more

Conditions: Retinitis Pigmentosa
Interventions: N-acetylcysteine, Placebo; Drug
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 485 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 24
States / cities: Davis, California • Los Angeles, California • San Francisco, California + 21 more

Conditions: Bardet-Biedl Syndrome
Interventions: Not listed
Lead sponsor: Marshfield Clinic Research Foundation; Other
Eligibility: Not listed
Enrollment: 1,200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2030
U.S. locations: 1
States / cities: Marshfield, Wisconsin

Conditions: Retinal Degeneration, Age-Related Macular Degeneration, Retinitis Pigmentosa, Hydroxychloroquine Retinopathy, Usher Syndromes, Late-Onset Retinal Degeneration, Cone Dystrophy, Cone Rod Dystrophy, Rod Cone Dystrophy, Rod Dystrophy
Interventions: Adaptive optics imaging; Device
Lead sponsor: Food and Drug Administration (FDA); Federal
Eligibility: 21 Years and older
Enrollment: 100 participants
Timeline: 2021 – 2028
U.S. locations: 2
States / cities: Bethesda, Maryland • Silver Spring, Maryland

Conditions: Retinitis Pigmentosa, Leber Congenital Amaurosis
Interventions: OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing; Drug
Lead sponsor: Ocugen; Industry
Eligibility: 6 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 7
States / cities: Phoenix, Arizona • La Jolla, California • Miami, Florida + 4 more

Conditions: Hepato/Renal Fibrocystic Disease, Autosomal Recessive Polycystic Kidney Disease, Joubert Syndrome, Bardet Biedl Syndrome, Meckel-Gruber Syndrome, Congenital Hepatic Fibrosis, Caroli Syndrome, Oro-Facial-Digital Syndrome Type I, Nephronophthisis, Glomerulocystic Kidney Disease
Interventions: Not listed
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Up to 18 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2030
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Boston, Massachusetts + 3 more

Conditions: Usher Syndrome, Retinitis Pigmentosa, Congenital Hearing Impairment
Interventions: Audiogram, Electroretinogram; Procedure
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2002
U.S. locations: 1
States / cities: New York, New York

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: QR-421a, Sham-procedure; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 12 Years and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 2
States / cities: Dallas, Texas • Madison, Wisconsin

Conditions: Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions: Arm 1; Procedure
Lead sponsor: MD Stem Cells; Industry
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2028
U.S. locations: 2
States / cities: Westport, Connecticut • Coral Springs, Florida

Conditions: Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Disease, Retinitis, Vision Tunnel, Vision Disorders
Interventions: QR-1123, Sham procedure; Drug · Other
Lead sponsor: ProQR Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 5
States / cities: Aurora, Colorado • Gainesville, Florida • Lexington, Kentucky + 2 more

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: AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 100 Years
Enrollment: 999 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2099
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions: Setmelanotide, Placebo; Drug
Lead sponsor: Rhythm Pharmaceuticals, Inc.; Industry
Eligibility: 6 Years and older
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 6
States / cities: San Diego, California • Springfield, Massachusetts • New York, New York + 3 more

Conditions: Retinitis Pigmentosa
Interventions: Not listed
Lead sponsor: National Eye Institute (NEI); NIH
Eligibility: Not listed
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1993 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome
Interventions: EA-2353; Drug
Lead sponsor: Endogena Therapeutics, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 5
States / cities: Miami, Florida • Ann Arbor, Michigan • Portland, Oregon + 2 more

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: QR-421a, Sham-procedure (dose cohort 1&2 only); Drug · Other
Lead sponsor: ProQR Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 4
States / cities: Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more

Conditions: Usher Syndrome, Retinitis Pigmentosa
Interventions: SAR421869; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 6 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2019
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD), Rod-Cone Dystrophy, Rod-Cone Disease, Retinal Degeneration, Cone-Rod Disease (C-RD), Cone-Rod Dystrophy
Interventions: OpCT-001; Biological
Lead sponsor: BlueRock Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 5
States / cities: Los Angeles, California • Miami, Florida • Philadelphia, Pennsylvania + 2 more

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: Ultevursen, Sham-procedure; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 12 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 10
States / cities: San Diego, California • San Francisco, California • Miami, Florida + 7 more

Conditions: Retinal Degeneration, Retinitis Pigmentosa
Interventions: Not listed
Lead sponsor: National Eye Institute (NEI); NIH
Eligibility: 4 Years and older
Enrollment: 3,549 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2016
U.S. locations: 3
States / cities: Bethesda, Maryland • Boston, Massachusetts • Cleveland, Ohio

Conditions: Retinitis Pigmentosa, Usher Syndrome Type 2
Interventions: RNA antisense oligonucleotide for intravitreal injection; Drug
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 12 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 4
States / cities: Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more

Conditions: Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease, Glaucoma
Interventions: RB (Retrobulbar), ST (Subtenon), IV (Intravenous), IVIT (Intravitreal), IO (Intraocular); Procedure
Lead sponsor: MD Stem Cells; Industry
Eligibility: 18 Years and older
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2020
U.S. locations: 1
States / cities: Westport, Connecticut

Conditions: Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions: Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention; Drug · Other
Lead sponsor: Laboratoires Thea; Industry
Eligibility: 8 Years and older
Enrollment: 81 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 10
States / cities: San Francisco, California • Miami, Florida • Atlanta, Georgia + 7 more

Conditions: Retinal Degeneration, Retinitis Pigmentosa, Eye Diseases, Hereditary
Interventions: Not listed
Lead sponsor: Jaeb Center for Health Research; Other
Eligibility: 8 Years and older
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 3
States / cities: San Francisco, California • Baltimore, Maryland • Durham, North Carolina

Search by objective public record fields.

Rate of Progression in USH2A-related Retinal Degeneration

Oral N-acetylcysteine for Retinitis Pigmentosa

Clinical Registry Investigating Bardet-Biedl Syndrome

Adaptive Optics Imaging of Outer Retinal Diseases

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

ARPKD Database Study

A Genetic Analysis of Usher Syndrome in Ashkenazi Jews

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)

Stem Cell Ophthalmology Treatment Study II

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

Inherited Retinal Degenerative Disease Registry

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

Study of Usher Syndromes, Type 1 and Type 2

Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

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)

Molecular Genetics of Retinal Degenerations

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa

Stem Cell Ophthalmology Treatment Study

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F