139 completed, 55 recruiting, 45 unknown, 34 active not recruiting. 82 Phase 2, 79 Phase 1, 78 Not applicable, 20 Phase 3, 7 Early Phase 1.
Follow Retinal Dystrophies: new trials by email Literature Review of Retinal Dystrophies trials Research Report on Retinal Dystrophies
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 |
|---|---|---|---|---|---|---|
| Non-invasive Ultrasound Retinal Stimulation for Vision Restoration Non-invasive ultrasound retinal stimulation Device - Age-Related Macular Degeneration, Retinitis Pigmentosa | Not applicable | Not yet recruiting | University of Southern California | 5 | 2027-01-10 | 2026-01-08 |
| 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 |
| Safety Evaluation Study of Pitavastatin PLGA Nano-particle for Retinitis Pigmentosa ULREA-PVS-NP - Retinitis Pigmentosa (RP) | Phase 1 | Recruiting | Kyushu University | 21 | 2026-10-01 | 2026-10-07 |
| Phase I/II Open-Label Trial of UGX202 for Advanced Retinitis Pigmentosa UGX202 Injection - Retinitis Pigmentosa (RP) | Phase 1 | Not yet recruiting | Suzhou UgeneX Therapeutics Co., Ltd. | 12 | 2026-09-28 | 2026-10-02 |
| Natural History Study of Advanced RCD Eye examinations, Questionnaire - Rod Cone Dystrophy | Not applicable | Recruiting | SparingVision | 100 | 2026-09-11 | 2026-10-08 |
| 9-cis Beta-Carotene-Rich Extract of Dunaliella Alga in Retinitis Pigmentosa Patients 9-cis beta-Carotene-rich extract of Dunaliella alga, Placebo Corn Oil - Retinitis Pigmentosa | Phase 1 / Phase 2 | Enrolling by invitation | Sheba Medical Center | 41 | 2026-07-26 | 2026-07-27 |
| Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment Active hf-tRNS., Placebo/sham hf-tRNS - Retinitis Pigmentosa (RP), Rod Cone Dystrophy | Not applicable | Recruiting | University of Waterloo | 20 | 2026-07-01 | 2026-07-07 |
| Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection) ZM-02 (low dose), ZM-02 (high dose), ZM-02 (selected dose) - Retinitis Pigmentosa (RP) | Phase 1 / Phase 2 | Not yet recruiting | Zhongmou Therapeutics | 21 | 2026-06-25 | 2026-04-13 |
| Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II 1.5 x 10^6 UC-MSC + CM, 5 x 10^6 UC-MSC + CM - Retinitis Pigmentosa | Phase 2 / Phase 3 | Not yet recruiting | PT. Prodia Stem Cell Indonesia | 30 | 2026-05-01 | 2026-02-25 |
| Long-term Safety of UC-MSC Transplantation in Patients With Retinitis Pigmentosa Conditioned Medium (CM), Umbilical Cord Mesenchymal Stem Cell (UC-MSC) - Retinitis Pigmentosa | Not yet recruiting | PT. Prodia Stem Cell Indonesia | 18 | 2026-05-01 | 2026-02-25 | |
| Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration PRIMA Products - Stargardt Disease, Retinitis Pigmentosa (RP) | Not applicable | Recruiting | Science Corporation | 5 | 2026-04-22 | 2026-08-17 |
| 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 | |
| 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 DSP-3077 Retinal Sheet Cohort 1, DSP-3077 Retinal Sheet Cohort 2, DSP-3077 Retinal Sheet Cohort 3 - Retinitis Pigmentosa | Phase 1 / Phase 2 | Recruiting | Sumitomo Pharma America, Inc. | 12 | 2026-04-16 | 2026-04-17 |
| A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) Phase 1b/2: Cohort 1 - OCT-980 Tablet, Phase 1b/2: Cohort 2 - OCT-980 Tablet, Phase 1b/2: Cohort 3 - OCT-980 Tablet - Retinitis Pigmentosa | Phase 1 / Phase 2 | Recruiting | Octant, Inc. | 18 | 2026-03-10 | 2026-09-24 |
| The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis Pigmentosa Alga Dunaliella Bardawil powder, Placebo:Starch - Retinitis Pigmentosa | Phase 1 / Phase 2 | Not yet recruiting | Sheba Medical Center | 30 | 2026-03-01 | 2025-10-06 |
| Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) Retinitis Pigmentosa, Stargardt Macular Dystrophy | Recruiting | Ray Therapeutics, Inc. | 25 | 2026-02-19 | 2026-03-31 | |
| Inherited Retinal Diseases: Natural History and Genotype-Phenotype Correlations No Intervention: Observational Cohort - Retinal Degenerations, Retinitis Pigmentosa (RP) | Not yet recruiting | IRCCS San Raffaele | 200 | 2026-01-01 | 2025-12-05 | |
| Wide Field OCTA in Ocular Diseases Age - Related Macular Degeneration (AMD), Diabetic Macular Edema | Not yet recruiting | IRCCS San Raffaele | 200 | 2026-01-01 | 2025-12-30 |
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Retinitis Pigmentosa (267) · Retinal Degeneration (43) · Cone-Rod Dystrophies (39) · Retinal Diseases (39) · Usher Syndromes (33) · Eye Diseases (32) · Macular Degeneration (31) · Stargardt Disease (21) · Eye Diseases, Hereditary (19) · Vision Disorders (18) · Choroideremia (15) · Glaucoma (14) · Diabetic Retinopathy (12) · Leber Congenital Amaurosis (12)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (306) · Genetic Diseases, Inborn (300) · Eye Diseases (292) · Eye Diseases, Hereditary (288) · Retinal Diseases (285) · Retinal Degeneration (281) · Nervous System Diseases (81) · Pathological Conditions, Signs and Symptoms (73) · Signs and Symptoms (71) · Neurologic Manifestations (67) · Sensation Disorders (58) · Congenital Abnormalities (44) · Abnormalities, Multiple (40) · Vision Disorders (38)
salicylhydroxamic acid (5) · Intravitreal Injections (4) · Ciliary Neurotrophic Factor (3) · Docosahexaenoic Acids (3) · Minocycline (3) · Vitamin A (3) · retinol acetate (3) · Acetylcysteine (2) · Culture Media, Conditioned (2) · Genotype (2) · Lutein (2) · Ophthalmic Solutions (2) · Tomography, Optical Coherence (2) · Valproic Acid (2) · Visual Prosthesis (2)
National Eye Institute (NEI) (16) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (11) · Johns Hopkins University (8) · Nanoscope Therapeutics Inc. (7) · Second Sight Medical Products (6) · Sheba Medical Center (6) · Ankara Universitesi Teknokent (5) · Columbia University (5) · Laboratoires Thea (5) · Okuvision GmbH (5) · Retina Implant AG (5) · SparingVision (5) · University of Michigan (5) · PYC Therapeutics (4) · QLT Inc. (4)
United States (139) · France (46) · Germany (36) · United Kingdom (31) · China (20) · Canada (19) · Italy (15) · Australia (14) · Netherlands (13) · Israel (12) · Brazil (9) · Switzerland (8) · Belgium (6) · Turkey (Türkiye) (6) · Austria (5)
Umut Arslan, MD (4) · Dr Samarendra Mohanty (3) · Jennifer Henrick, RN, MS (3) · Mohamed Genead, MD (3) · Rubens C Siqueira, MD,PhD (3) · Sushanta Mallick (3) · Anne-Marie Ripley (2) · Brett G Jeffrey, Ph.D. (2) · Brian P Brooks, M.D. (2) · David Liao, MD (2) · Florian Gekeler, Prof Dr med (2)
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