10 recruiting, 7 completed, 2 active not recruiting, 2 unknown. 22 Phase 1, 13 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 22 | Recruiting 10, Completed 7, Active not recruiting 2, Unknown 2, Terminated 1 |
| Phase 2 | 13 | Recruiting 5, Completed 4, Active not recruiting 2, Terminated 1, Unknown 1 |
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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 |
|---|---|---|---|---|---|---|
| A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants CITY-RBP4, Placebo - Stargardt Disease 1, STGD1 | Phase 1 | Recruiting | City Therapeutics | 48 | 2026-09-01 | 2026-09-23 |
| A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions ASP2020 - Stargardt Disease, Stargardt Macular Dystrophy | Phase 1 | Recruiting | Astellas Institute for Regenerative Medicine | 30 | 2026-08-14 | 2026-10-28 |
| Phase 1 Study of C.001 in Retinal Degeneration Injection - Geographic Atrophy, Stargardt Disease | Phase 1 | Recruiting | Cellio Therapeutics Inc | 15 | 2026-04-07 | 2026-08-17 |
| Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease RTx-021 - Stargardt Disease | Phase 1 / Phase 2 | Recruiting | Ray Therapeutics, Inc. | 18 | 2026-01-28 | 2026-04-15 |
| A Study of DC6001 Tablet in Healthy Chinese Adult Subjects DC6001, Placebo - Stargardt Disease | Phase 1 | Recruiting | Heronova Pharmaceuticals | 76 | 2026-01-01 | 2026-02-18 |
| A Study of AAVB-039 in Participants With Stargardt Disease (STGD1) AAVB-039 - Stargardt Disease | Phase 1 / Phase 2 | Recruiting | AAVantgarde Bio Srl | 75 | 2025-09-29 | 2026-09-30 |
| The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration Oral disulfiram - Alcohol Use Disorder, Retinal Dystrophies | Phase 1 | Recruiting | University of Rochester | 15 | 2025-05-19 | 2026-09-16 |
| A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1) SB-007 - Stargardt Disease, Stargardt Macular Degeneration | Phase 1 / Phase 2 | Recruiting | Splice Bio | 57 | 2025-02-11 | 2025-04-29 |
| Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease) VG801 - Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1 | Phase 1 / Phase 2 | Recruiting | VeonGen Therapeutics GmbH | 18 | 2024-12-23 | 2026-09-02 |
| Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR) ACDN-01 - Stargardt Disease, Cone Rod Dystrophy | Phase 1 / Phase 2 | Recruiting | Ascidian Therapeutics, Inc | 15 | 2024-06-11 | 2026-10-07 |
| Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1) JWK006 - Stargardt Disease 1 | Phase 1 / Phase 2 | Active not recruiting | West China Hospital | 9 | 2023-11-20 | 2024-03-08 |
| This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease tinlarebant - Stargardt Disease | Phase 1 / Phase 2 | Completed | RBP4 Pty Ltd | 13 | 2021-03-12 | 2024-04-12 |
| Oral Metformin for Treatment of ABCA4 Retinopathy Metformin hydrochloride - ABCA4 Retinopathy, Stargardt Disease | Phase 1 / Phase 2 | Active not recruiting | National Eye Institute (NEI) | 55 | 2020-11-23 | 2026-10-22 |
| Stem Cells Therapy in Degenerative Diseases of the Retina Stem/progenitor cells transplantation - Retinal Degeneration, Retinitis Pigmentosa | Phase 1 | Unknown | Pomeranian Medical University Szczecin | 30 | 2018-12-13 | 2018-12-17 |
| Stem Cell Therapy for Outer Retinal Degenerations injection of hESC-RPE in suspension, injection hESC-RPE seeded in a substrate - Age Related Macular Degeneration, Stargardt's Disease | Phase 1 / Phase 2 | Completed | Federal University of São Paulo | 15 | 2015-08-01 | 2020-07-22 |
| Phase 1 Safety Study of ALK-001 in Healthy Volunteers ALK-001 (No generic name) - Stargardt Disease, Age-related Macular Degeneration | Phase 1 | Completed | Alkeus Pharmaceuticals, Inc. | 40 | 2014-04-01 | 2025-04-27 |
| Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD) MA09-hRPE - Stargardt's Macular Dystrophy | Phase 1 / Phase 2 | Completed | Astellas Institute for Regenerative Medicine | 12 | 2011-12-13 | 2024-10-31 |
| Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy MA09-hRPE - Stargardt's Macular Dystrophy | Phase 1 / Phase 2 | Completed | Astellas Institute for Regenerative Medicine | 13 | 2011-06-16 | 2024-10-31 |
| Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration SAR422459 - Stargardt's Disease | Phase 1 / Phase 2 | Terminated | Sanofi | 27 | 2011-06-08 | 2022-04-14 |
| Saffron Supplementation in Stargardt's Disease Saffron supplementation, placebo - Retinal Degeneration, Genetic Disease | Phase 1 / Phase 2 | Unknown | Catholic University of the Sacred Heart | 30 | 2011-02-01 | 2017-07-19 |
Showing 20 of 22. Sign in to see all of them.
Macular Degeneration (9) · Retinal Degeneration (4) · Retinal Diseases (2) · Retinal Dystrophies (2) · Retinitis Pigmentosa (2) · Alcoholism (1) · Cone-Rod Dystrophies (1) · Corneal Dystrophies, Hereditary (1) · Erythrokeratodermia with ataxia (1) · Eye Diseases (1) · Genetic Diseases, Inborn (1) · Geographic Atrophy (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (22) · Eye Diseases, Hereditary (22) · Eye Diseases (21) · Genetic Diseases, Inborn (21) · Retinal Diseases (20) · Retinal Degeneration (18) · Macular Degeneration (13) · Retinal Dystrophies (2) · Alcohol-Related Disorders (1) · Chemically-Induced Disorders (1) · Corneal Diseases (1) · Mental Disorders (1) · Substance-Related Disorders (1)
Dietary Supplements (1) · Disulfiram (1) · Docosahexaenoic Acids (1) · Fomepizole (1) · Injections (1) · Metformin (1) · Sodium Chloride (1) · Suspensions (1)
Astellas Institute for Regenerative Medicine (3) · National Eye Institute (NEI) (2) · AAVantgarde Bio Srl (1) · Alkeus Pharmaceuticals, Inc. (1) · Ascidian Therapeutics, Inc (1) · Catholic University of the Sacred Heart (1) · Cellio Therapeutics Inc (1) · City Therapeutics (1) · Federal University of São Paulo (1) · Heronova Pharmaceuticals (1) · Pomeranian Medical University Szczecin (1) · RBP4 Pty Ltd (1) · Ray Therapeutics, Inc. (1) · Sanofi (1) · Splice Bio (1)
United States (12) · China (3) · Australia (2) · United Kingdom (2) · Brazil (1) · France (1) · Italy (1) · Norway (1) · Poland (1) · Taiwan (1)
Benedetto Falsini, MD (1) · Brian P Brooks, M.D. (1) · Fang Lu, MD (1) · Jose-Alain Sahel, MD. Ph.D (1) · Marco Piccardi, MD (1) · Murray Abramson, MD, PhD (1) · Paul S Bernstein, M.D., Ph.D. (1) · Paul Yang, MD (1) · Rubens Belfort, MD (1) · Silvia Bisti, PhD (1) · Xiaodong Sun, MD (1)
PMID 30194931, PMID 17963111, PMID 22281388, PMID 25458728, PMID 27730010, PMID 29884405, PMID 31618812, PMID 39281698