Table 2.
Treatments for Stargardt Disease Registered on clinicaltrials.gov (February 2025)
| Treatment Type | Intervention and Comparator | Study Design | Clinical Trial Information | Sponsor |
|---|---|---|---|---|
| Pharmacological therapies targeting toxic bis-retinoids | ||||
| Gildeuretinol | ALK-001, oral administration for 24 mo (Comparator: placebo) | Placebo-controlled RCT | NCT02402660 (TEASE, Phase II, recruitment completed); NCT04239625 (Open label extension) | Alkeus Pharmaceuticals, Inc |
| Tinlarebant (RBP4 antagonist) | LBS-008, oral administration of 5 mg/day for 24 mo (Comparator: placebo) | Placebo-controlled RCT | NCT06388083 (Phase II/III, recruiting); NCT05244304 (Phase III, recruitment completed) | Belite Bio, Inc |
| STG-001 (RBP4 antagonist) | STG-001 oral administration, (two dosage groups), for 28 days | Open-label, non-randomized study | NCT04489511 (Phase II, completed 2024, results submitted online) | Stargazer Pharmaceuticals, Inc |
| Emixustat | Emixustat hydrochloride, ACU-4429, oral administration of 10 mg/day in the Phase III trial (Comparator: placebo) | Placebo-controlled RCT | NCT03033108 (Phase II, completed in 2017);75 NCT03772665 (Phase III [SeaSTAR], completed in 2022, results posted online) | Kubota Vision Inc |
| 4-methylpyrazole | 4-methylpyrazole (4-MP, fomepizole, Antizol) infusion 15 mg/kg dose (Comparator: saline) | Placebo-controlled RCT | NCT00346853 (Phase I, completed in 2006, no results posted) | University of Utah |
| Gene therapy, optogenetics, and RNA therapy | ||||
| RORA adeno-associated virus vector gene therapy | OCU410ST single subretinal injection | Open-Label, non-randomized, dose escalation study (Phase I) and randomized dose expansion study (Phase II) | NCT05956626 (GARDian, Phase I/II, recruiting) | Ocugen |
| Adeno-associated virus vector gene therapy | JWK006 single subretinal injection | Open-Label, non-randomized, single ascending dose study | NCT06300476 (phase I/II, active, not recruiting) | West China Hospital |
| Lentivirus (Equine infectious anemia virus (EIAV)) | SAR422459 single subretinal injection | Open-Label, non-randomized, single ascending dose study | NCT01367444 (Phase I/II, terminated); NCT01736592 (follow-up study of the 27 participants enrolled) | Sanofi |
| Optogenetics Therapy | vMCO-010 (1.2E11gc/eye) multi-Characteristic Opsin single intravitreal injection | Open-label, non-randomized trial | NCT05417126 (STARLIGHT, Phase 2, completed 2023, results submitted online) | Nanoscope Therapeutics Inc |
| RNA exon editor | ACDN-01, single subretinal injection (3 doses) | Open-Label, non-randomized, single ascending dose study | NCT06467344 (STELLAR, Phase I/II, recruiting) | Ascidian Therapeutics, Inc |
| Stem cell therapy | ||||
| hESC-derived retinal pigment epithelium cells RPE cells | MA09-hRPE, single subretinal injection (4 dose groups between 50,000–200,000 cells) | Open-Label, non-randomized trial | NCT01345006, NCT01469832 (Phase I/II, completed in 2015);76 NCT02445612, NCT02941991 (follow-up study, completed 2019) | Astellas Institute for Regenerative Medicine |
| hESC-derived retinal pigment epithelium cells | MA09-hRPE single subretinal injection with 50000 cells | Open-Label, non-randomized trial | NCT01625559 (Phase I, completed in 2015)77 | CHABiotech CO., Ltd |
| hESC-derived retinal pigment epithelium cells | hESC-RPE, delivered as either a single subretinal injection with 100,000 cells, or subretinal implantation seeded in a monolayer in a polymeric substrate | Open-Label, non-randomized trial | NCT02903576 (Phase I/II, completed in 2019) | Federal University of São Paulo |
| Bone marrow-derived stem/progenitor cells | Autologous bone marrow derived stem cells (BMSC), delivered as retrobulbar, subtenons, intravitreal or subretinal and intravenous injections | Open-Label, non-randomized trial | NCT01920867 (SCOTS, completed in 2020);78 NCT03011541 (SCOTS2, recruiting) | MD Stem Cells |
| bone marrow-derived stem/progenitor cells | Autologous bone marrow stem/progenitor cell intravitreal injection in enrolled patients by repeated follow-up over 1 y with clinical evaluations | Open-Label, non-randomized trial | NCT03772938 (Phase I, status unknown, no results posted) | Pomeranian Medical University Szczecin |
| Other therapies | ||||
| Complement C5 Inhibitor | Avacincaptad pegol (Zimura) intravitreal injection monthly for monthly for up to 17 mo (Comparator: sham injection) | Placebo-controlled RCT | NCT03364153 (Phase II, active, not recruiting) | Astellas Pharma Global Development, Inc |
| Saffron | Saffron oral supplementation 20 mg/day (Comparator: placebo) | cross-over Placebo-controlled RCT | NCT01278277 (Phase I/II cross-over RCT)79 | Catholic University of the Sacred Heart |
| Omega-3 fatty acid | Combined long-chain omega-3 fatty acid (3660 mg/day eicosapentaenoic acid [EPA] and docosahexaenoic acid [DHA]) for 24 weeks (Comparator: placebo (sunflower oil)) | Placebo-controlled RCT | NCT03297515 (MADEOS, completed)80 | Ophthalmos Research and Education Institute |
| Omega-3 fatty acid (docosahexaenoic acid) | Docosahexaenoic acid oral supplements (2000 mg/day) for 3 mo (Comparator: placebo (corn oil and soy oil)) | Cross-over Placebo-controlled RCT | NCT00060749 (Phase I, completed in 2007)74 | National Eye Institute (NEI) |
| Metformin | Metformin oral administration of metformin of 500 mg/day at study entry and titrated up to reach 2000 mg/day | Open label, single group treatment | NCT04545736 (Phase I/II, recruiting) | National Institutes of Health Clinical Center (CC) |
| Disulfiram | Disulfiram (Antabuse) oral administration 250 mg/day | Open label, single group treatment | NCT06319872 (Phase I, recruiting) | University of Rochester |
| Acupuncture | Acupuncture and massage therapy (initially once/week for 24 mo) | Open-label, single group treatment | NCT02255981 (completed, results posted online) | Escuela Neijing |
| Microcurrent stimulation therapy | Microcurrent Stimulation Therapy, no further information | Not specified | NCT01790958 (Phase I, completed in 2012, no results posted) | Retina Institute of Hawaii |
RCT, randomized controlled trial.