11 completed, 9 recruiting, 5 terminated, 3 active not recruiting. 12 Phase 2, 6 Phase 1, 5 Phase 3, 2 Not applicable.
Follow Usher Syndromes: new trials by email Literature Review of Usher Syndromes trials Research Report on Usher Syndromes
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome N-acetylcysteine amide, Placebo - Retinitis Pigmentosa (RP), Usher Syndrome | Phase 3 | Not yet recruiting | Nacuity Pharmaceuticals, Inc. | 80 | 2026-12-01 | 2026-07-01 |
| 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 No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen - Retinitis Pigmentosa (RP), Usher Syndrome Type 2 | Phase 2 | Not yet recruiting | Laboratoires Thea | 81 | 2026-10-05 | 2026-09-01 |
| A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome Placebo, NPI-001 - Retinitis Pigmentosa (RP), Usher Syndrome | Phase 3 | Recruiting | Nacuity Pharmaceuticals, Inc. | 80 | 2026-10-01 | 2026-10-13 |
| Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance Transcorneal Electrical Stimulation using the OkuStim System - Retinitis Pigmentosa (RP), Usher Syndrome | Recruiting | Okuvision GmbH | 30 | 2026-04-20 | 2026-05-01 | |
| Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7A Vision tests, Retinal imaging, Questionnaires - Usher Syndrome | Recruiting | Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts | 60 | 2025-10-13 | 2026-02-10 | |
| A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO) OpCT-001 - Primary Photoreceptor Disease, Retinitis Pigmentosa (RP) | Phase 1 / Phase 2 | Recruiting | BlueRock Therapeutics | 54 | 2025-03-10 | 2026-10-15 |
| Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention - Retinitis Pigmentosa (RP), Usher Syndrome Type 2 | Phase 2 | Active not recruiting | Laboratoires Thea | 81 | 2024-12-11 | 2026-07-09 |
| BF844 Safety and Pharmacokinetic Study in Healthy Volunteers BF844 - Usher Syndrome Type 3 | Phase 1 | Unknown | EyeXCel Pty. Ltd. | 76 | 2024-09-09 | 2024-09-19 |
| Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa AAVB-081 - Usher Syndrome, Type 1B | Phase 1 / Phase 2 | Recruiting | AAVantgarde Bio Srl | 15 | 2024-07-02 | 2025-05-02 |
| Oral N-acetylcysteine for Retinitis Pigmentosa N-acetylcysteine, Placebo - Retinitis Pigmentosa | Phase 3 | Active not recruiting | Johns Hopkins University | 485 | 2023-10-11 | 2026-06-15 |
| 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) QR-421a, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 5 | 2021-12-15 | 2024-08-07 |
| 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) Ultevursen, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 7 | 2021-12-08 | 2024-07-12 |
| Auditory Neural Function in Implanted Patients With Usher Syndrome Experimental manipulation of stimulation parameters - Usher Syndrome, Cochlear Implantation | Not applicable | Completed | Ohio State University | 29 | 2021-10-01 | 2025-07-29 |
| An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa RNA antisense oligonucleotide for intravitreal injection - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 | Terminated | Laboratoires Thea | 21 | 2021-09-16 | 2024-08-07 |
| Adaptive Optics Imaging of Outer Retinal Diseases Adaptive optics imaging - Retinal Degeneration, Age-Related Macular Degeneration | Recruiting | Food and Drug Administration (FDA) | 100 | 2021-08-27 | 2026-05-08 | |
| Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome NPI-001, Placebo - Usher Syndromes | Phase 1 / Phase 2 | Completed | Nacuity Pharmaceuticals, Inc. | 49 | 2020-09-03 | 2026-07-01 |
| Prospective Open Clinical and Genetic Study of Patients With Retinitis Pigmentosa Whole Exome Sequencing - Retinitis Pigmentosa, Usher Syndromes | Completed | Sensor Technology for Deafblind | 130 | 2019-03-26 | 2022-05-20 | |
| Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene QR-421a, Sham-procedure (dose cohort 1&2 only) - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 1 / Phase 2 | Completed | ProQR Therapeutics | 20 | 2019-03-06 | 2022-04-20 |
| Natural History Study of Usher Syndrome ( Light4Deaf ) Usher Syndromes | Recruiting | Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts | 400 | 2017-06-08 | 2020-12-14 | |
| Clinical and Genetic Testing of Patients With Usher Syndrome Usher Syndrome, Congenital Deafness | Completed | Sensor Technology for Deafblind | 28 | 2017-05-17 | 2019-04-03 |
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Retinitis Pigmentosa (26) · Retinal Diseases (9) · Vision Disorders (7) · Eye Diseases (6) · Eye Diseases, Hereditary (6) · Cone-Rod Dystrophies (5) · Eye Abnormalities (5) · Cone Dystrophy (4) · Retinal Degeneration (4) · Retinal Dystrophies (3) · Blindness (2) · Deafness (2) · Genetic Diseases, Inborn (2) · Macular Degeneration (2)
Abnormalities, Multiple (33) · Congenital Abnormalities (33) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (33) · Deaf-Blind Disorders (33) · Ear Diseases (33) · Hearing Loss, Sensorineural (33) · Nervous System Diseases (33) · Neurologic Manifestations (33) · Otorhinolaryngologic Diseases (33) · Pathological Conditions, Signs and Symptoms (33) · Sensation Disorders (33) · Signs and Symptoms (33) · Hearing Disorders (32) · Hearing Loss (32) · Blindness (31)
Intravitreal Injections (4) · Acetylcysteine (1) · Blood Specimen Collection (1) · Ciliary Neurotrophic Factor (1) · Electroconvulsive Therapy (1) · Exome (1) · Genotype (1) · Hearing (1) · N-Acetylcysteinamide (1) · Optical Imaging (1) · Vision Tests (1) · salicylhydroxamic acid (1)
Laboratoires Thea (5) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (3) · Nacuity Pharmaceuticals, Inc. (3) · National Eye Institute (NEI) (3) · National Center for Research Resources (NCRR) (2) · Sanofi (2) · Sensor Technology for Deafblind (2) · AAVantgarde Bio Srl (1) · BlueRock Therapeutics (1) · EyeXCel Pty. Ltd. (1) · Food and Drug Administration (FDA) (1) · Foundation Fighting Blindness (1) · Johns Hopkins University (1) · MD Stem Cells (1) · MejoraVisionMD (1)
United States (20) · France (8) · Germany (5) · United Kingdom (5) · Canada (4) · Netherlands (4) · Australia (2) · Austria (2) · Italy (2) · Pakistan (2) · Russia (2) · Belgium (1) · Brazil (1) · China (1) · Denmark (1)
Benedict Tan, MBBS, FRACP (1) · Daniel X Hammer, Ph.D. (1) · Dmitry S. Atarshchikov, MD, PhD (1) · Isabelle Audo, Pr (1) · Jacque Duncan, MD (1) · James F Hejtmancik, M.D. (1) · Jeffrey Weiss, MD (1) · Jose-Alain Sahel, MD, PhD (1) · Lee Anderson, MD (1) · Peter A Campochiaro, MD (1)
PMID 24705452, PMID 26199618, PMID 26604914, PMID 27857759, PMID 27904503, PMID 29270420, PMID 30050918, PMID 30358468, PMID 31119146, PMID 31215818, PMID 31620478, PMID 32039263, PMID 32231088, PMID 32573764, PMID 33546345, PMID 37418643, PMID 40339601, PMID 41282901, PMID 42635483
1UG1EY033293 (1), 2018 (1), R21DC019458 (1)