24 completed, 22 recruiting, 10 active not recruiting, 4 unknown. 22 Phase 1, 22 Phase 2, 8 Not applicable, 5 Phase 3, 1 Early Phase 1.
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 |
|---|---|---|---|---|---|---|
| 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 | 2026-10-23 |
| Study of ALK-001 on the Progression of Stargardt Disease ALK-001, Placebo - Stargardt Disease | Phase 3 | Recruiting | Alkeus Pharmaceuticals, Inc. | 230 | 2026-06-03 | 2026-09-18 |
| 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 |
| 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 |
| A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults No Intervention - Stargardt Disease, Stargardt Macular Dystrophy | Recruiting | Astellas Pharma Global Development, Inc. | 90 | 2026-02-20 | 2026-10-20 | |
| 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 | |
| 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 |
| 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 | |
| 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 |
| 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 | |
| Feasibility and Tolerability Study of Smart Contact Lens With Healthy Subjects and Patients With Stargardt's Disease SCL miniaturized scleral lens positioning - Stargardt Disease | Not applicable | Not yet recruiting | Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts | 32 | 2025-10-01 | 2025-09-18 |
| 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 |
| An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease HG005 - Stargardt Disease Type 1 (STGD1) | Early Phase 1 | Recruiting | HuidaGene Therapeutics Co., Ltd. | 6 | 2025-08-20 | 2025-08-27 |
| 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 |
| A Prospective Observational Study to Assess the Reliability and Validity of the MLSDT Observational - Macular Degeneration, Geographic Atrophy | Recruiting | Nanoscope Therapeutics Inc. | 30 | 2025-01-28 | 2025-03-24 | |
| 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 |
| Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH) Prescreening Assessments - Stargardt Disease, Stargardt Disease 1 | Recruiting | Ascidian Therapeutics, Inc | 50 | 2024-06-20 | 2026-10-09 |
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Macular Degeneration (19) · Retinitis Pigmentosa (15) · Retinal Degeneration (9) · Retinal Dystrophies (9) · Retinal Diseases (8) · Cone-Rod Dystrophies (5) · Eye Diseases (5) · Geographic Atrophy (5) · Choroideremia (3) · Cone Dystrophy (3) · Eye Diseases, Hereditary (3) · Vision, Low (3) · Vitelliform Macular Dystrophy (3) · Diabetic Retinopathy (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (66) · Genetic Diseases, Inborn (65) · Eye Diseases, Hereditary (63) · Eye Diseases (61) · Retinal Diseases (58) · Retinal Degeneration (57) · Macular Degeneration (47) · Retinal Dystrophies (12) · Nervous System Diseases (7) · Cardiovascular Diseases (5) · Metabolic Diseases (5) · Nutritional and Metabolic Diseases (5) · Cranial Nerve Diseases (4) · Genetic Diseases, X-Linked (4) · Neurologic Manifestations (4)
emixustat (2) · Acupuncture Therapy (1) · Dietary Supplements (1) · Disulfiram (1) · Docosahexaenoic Acids (1) · Fomepizole (1) · Immunoglobulins, Intravenous (1) · Injections (1) · Jupiter (1) · Massage (1) · Medicine, Chinese Traditional (1) · Metformin (1) · Paracentesis (1) · Sodium Chloride (1) · Suspensions (1)
Astellas Institute for Regenerative Medicine (5) · Alkeus Pharmaceuticals, Inc. (4) · Nanoscope Therapeutics Inc. (3) · National Eye Institute (NEI) (3) · Ray Therapeutics, Inc. (3) · AAVantgarde Bio Srl (2) · Ascidian Therapeutics, Inc (2) · Astellas Pharma Global Development, Inc. (2) · Belite Bio, Inc (2) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (2) · Foundation Fighting Blindness (2) · IRCCS San Raffaele (2) · Johns Hopkins University (2) · Kubota Vision Inc. (2) · MD Stem Cells (2)
United States (42) · United Kingdom (11) · France (9) · Germany (8) · China (6) · Italy (5) · Australia (4) · Spain (3) · Brazil (2) · Netherlands (2) · Norway (2) · Switzerland (2) · Taiwan (2) · Austria (1) · Belgium (1)
Jeffrey Weiss, MD (2) · Murray Abramson, MD, PhD (2) · Steven Levy, MD (2) · Benedetto Falsini, MD (1) · Brett G Jeffrey, Ph.D. (1) · Brian P Brooks, M.D. (1) · Dr Samarendra Mohanty (1) · Dr. Samarendra Mohanty (1) · Fang Lu, MD (1) · George Papastergiou, MD (1) · Hendrik Scholl, MD (1)
PMID 22281388, PMID 25458728, PMID 26199618, PMID 26604914, PMID 27857759, PMID 27904503, PMID 30194931, PMID 17963111, PMID 25104651, PMID 26265646, PMID 26786511, PMID 27378015, PMID 27730010, PMID 28549516, PMID 28930518, PMID 29049437, PMID 29270420, PMID 29311565, PMID 29884405, PMID 29902293, PMID 30050918, PMID 31119146, PMID 31266057, PMID 31583549, PMID 31618812, PMID 31620478, PMID 32039263, PMID 32231088, PMID 32568958, PMID 33073619, PMID 33214244, PMID 33546345, PMID 34803584, PMID 35196835, PMID 39281698, PMID 42690655, PMID 9111255
2017 (1)