8 completed, 7 active not recruiting, 5 recruiting, 1 terminated. 22 Phase 2, 13 Phase 1, 2 Phase 3.
Follow Stargardt Disease: new trials by email Literature Review of Stargardt Disease trials Research Report on Stargardt Disease
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 |
|---|---|---|---|---|---|---|
| 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 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 |
| 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 |
| A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease Tinlarebant, Placebo - STGD1, Stargardt Disease 1 | Phase 2 / Phase 3 | Active not recruiting | Belite Bio, Inc | 60 | 2024-07-31 | 2026-03-12 |
| 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 |
| A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease OCU410ST - Stargardt Disease | Phase 2 / Phase 3 | Active not recruiting | Ocugen | 51 | 2023-08-25 | 2026-07-31 |
| Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease Gene Therapy-vMCO-010 - Stargardt Disease | Phase 2 | Completed | Nanoscope Therapeutics Inc. | 6 | 2022-07-05 | 2023-11-09 |
| 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 |
| Study of STG-001 in Subjects With Stargardt Disease STG-001 - Stargardt Disease-1 | Phase 2 | Completed | Stargazer Pharmaceuticals, Inc. | 10 | 2020-10-01 | 2021-04-27 |
| Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) ALK-001 - Stargardt Disease, Stargardt Macular Degeneration | Phase 2 | Active not recruiting | Alkeus Pharmaceuticals, Inc. | 200 | 2019-12-20 | 2026-09-21 |
| Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1) avacincaptad pegol, Sham - Stargardt's Macular Dystrophy | Phase 2 | Completed | Astellas Pharma Global Development, Inc. | 121 | 2018-01-12 | 2026-04-15 |
| Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease Emixustat - Stargardt Disease, Macular Atrophy | Phase 2 | Completed | Kubota Vision Inc. | 23 | 2017-01-01 | 2021-05-19 |
| Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease ALK-001, Placebo - Stargardt Disease, Stargardt Macular Degeneration | Phase 2 | Active not recruiting | Alkeus Pharmaceuticals, Inc. | 160 | 2015-08-01 | 2026-09-21 |
| 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 I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration Long term follow up in all patients who received SAR422459 in previous study TDU13583 - Stargardt's Disease | Phase 2 | Active not recruiting | Sanofi | 27 | 2012-12-14 | 2026-08-26 |
| 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 |
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Retinal Degeneration (4) · Macular Degeneration (3) · Eye Diseases (2) · Retinal Diseases (2) · Retinal Dystrophies (2) · Anetoderma (1) · Cone-Rod Dystrophies (1) · Eye Diseases, Hereditary (1) · Genetic Diseases, Inborn (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (22) · Eye Diseases, Hereditary (21) · Genetic Diseases, Inborn (21) · Eye Diseases (20) · Retinal Diseases (20) · Macular Degeneration (19) · Retinal Degeneration (18) · Connective Tissue Diseases (1) · Retinal Dystrophies (1) · Skin Abnormalities (1) · Skin Diseases (1) · Skin and Connective Tissue Diseases (1)
Metformin (1) · Suspensions (1) · emixustat (1) · salicylhydroxamic acid (1)
Alkeus Pharmaceuticals, Inc. (2) · Astellas Institute for Regenerative Medicine (2) · Sanofi (2) · AAVantgarde Bio Srl (1) · Ascidian Therapeutics, Inc (1) · Astellas Pharma Global Development, Inc. (1) · Belite Bio, Inc (1) · Catholic University of the Sacred Heart (1) · Federal University of São Paulo (1) · Kubota Vision Inc. (1) · Nanoscope Therapeutics Inc. (1) · National Eye Institute (NEI) (1) · Ocugen (1) · RBP4 Pty Ltd (1) · Ray Therapeutics, Inc. (1)
United States (16) · United Kingdom (4) · France (3) · China (2) · Italy (2) · Australia (1) · Brazil (1) · Germany (1) · Hungary (1) · Israel (1) · Japan (1) · Norway (1) · Spain (1) · Taiwan (1)
Benedetto Falsini, MD (1) · Brian P Brooks, M.D. (1) · Dr Samarendra Mohanty (1) · Fang Lu, MD (1) · Jennifer Henrick, RN, MS (1) · Jose-Alain Sahel, MD. Ph.D (1) · Marco Piccardi, MD (1) · Mohamed Genead, MD (1) · Murray Abramson, MD, PhD (1) · Paul Yang, MD (1) · Rubens Belfort, MD (1) · Silvia Bisti, PhD (1)
PMID 30194931, PMID 22281388, PMID 25458728, PMID 27730010, PMID 29884405, PMID 31618812, PMID 33214244, PMID 39281698, PMID 42690655
2017 (1)