On this page you can find a list of clinical trials for inherited retinal diseases, such as retinitis pigmentosa and Stargardt disease, as well as for some innovative treatments for age-related macular degeneration (AMD) and diabetes-related eye conditions.

Where available, the following information is included for each trial:

• Type of treatment, such as gene therapy, stem cell therapy or a drug
• Gene and mutation, or cell type
• Drug name (alternative or brand name in brackets)
• Trial sponsor and phase (TRIAL NAME capitalized in brackets)
• Other information including if there is a Canadian trial site, or links to completed trial results or updates
• Clinicaltrial.gov number. This will link out to trial page on the clinicaltrials.gov website

You can find more information about these or other clinical trials at ClinicalTrials.gov. This website can be a good place to start your research. However, trials listed have not always been approved by the FDA and may not meet basic safety standards. Always consult your doctor before participating in any trial.

ACHROMATOPSIA

• Drug: glycerol phenylbutyrate (PBA), for patients with mutations in ATF6, Columbia University, Early Phase 1, NCT04041232
• Gene therapy: CNGA3 (AGTC-402), Beacon Therapeutics, Phase 1/2, Discontinued (2023), NCT02935517
• Gene therapy: CNGB3 (AGTC-401), Beacon Therapeutics, Phase 1/2, Discontinued (2023), NCT02599922
• Gene therapy: CNGA3, STZ eye trial (COLOURBRIDGE), Phase 1/2, Update (2021), NCT02610582

AGE-RELATED MACULAR DEGENERATION (AMD)

Neovascular (wet) AMD

• Cell therapy: RPE (PF-05206388), Moorfields Eye Hospital NHS Foundation Trust, Phase 1, NCT01691261
• Drug: AXT107 (DISCOVER). AsclepiX Therapeutics, Phase 1/2, Update (2024), NCT05859776
• Gene therapy: anti-VEGF (RGX-314) AbbVie: Phase 2 (AAVIATE), Update (2024) (2022), NCT04514653
• Gene therapy: anti-VEGF (RGX-314) AbbVie: Phase 2/3 (ATMOSPHERE) NCT04704921, Phase 3 (ASCENT), NCT05407636
• Gene Therapy: anti-VEGF (ADVM-022, Ixo-vec), Adverum Biotechnologies (LUNA), Phase 2, Update (2024), (2023), NCT05536973
• Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1, Update (2022), Completed, NCT03585556
• Gene Therapy: anti-VEGF and VEGF-C RNAi, 4D-150, 4D Molecular Therapeutics, Phase 1/2, Update (2024), (2022), NCT05197270

Geographic atrophy (Advanced Dry AMD)

• Bionics: PRIMA System, Pixium Vision, Update (2022), NCT03392324
• Cell therapy : RPE (ASP7317), Astellas, Phase 1, NCT03178149
• Cell therapy: RPE cells (OpRegen), Genetech/Roche, Phase 2, Update (2023), NCT05626114
• Cell therapy : RPE, NEI, Phase 1/2, Update (2024), NCT04339764
• Cell therapy : RPE (CPCB-RPE1), Regenerative Patch Technologies, Phase 1/2, Update (2022), NCT02590692.
• Drug: ALK-001 (SAGA), Alkeus Pharmaceuticals, Phase 3, NCT03845582
• Drug: Ionis-FB-LRx (Complement Factor B inhibitor), Ionis (GOLDEN), Phase 2, Canadian trial sites, Completed, NCT03815825
• Drug: Pegcetacoplan (Syfovre, Complement C3 inhibitor), Apellis (GALE), Phase 3, Complete (2022), NCT04770545. Approved by FDA 2023
• Drug: Tinlarebant (PHOENIX), Belite Bio, Phase 3, Update (2024), NCT05949593
• Drug: Zimura (Complement C5 inhibitor), IVERIC bio (GATHER1), Phase 3, Complete (2023), NCT04435366. Acquired by Astellas and approved by FDA 2023.
• Gene therapy: OCU410 (ArMaDa), Ocugen, Phase 1/2, NCT06018558
• Gene therapy: GT005 (HORIZON, EXPLORE), Gyroscope/Novartis, Phase 2, Discontinued (2023),  NCT04566445, NCT04437368
• Photobiomodulation: LumiThera, Update (2024), (2022), NCT04065490  

CHOROIDEREMIA

• Gene therapy: REP1 (4D-110), 4D Molecular Therapeutics, Phase 1, Update (2023), NCT04483440
• Gene therapy: REP1 (BIIB111), NightstaRx/Biogen (STAR), Phase 3, Canadian trial sites, Completed, NCT03496012
• Gene therapy: REP1 (SPK-7001), Spark Therapeutics, Phase 1/2, Completed, NCT02341807
• Gene therapy: REP1, STZ eye trial (THOR), Phase 2, Completed, NCT02671539
• Gene therapy: REP1, University of Oxford (REGENERATE), Phase 2, Completed, NCT02407678
• Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, Update (2024), (2023), NCT05282953

Diabetes-related Eye Conditions

• Drug: EXN407, Exonate Limited, Phase 1/2, Update (2023), NCT04565756
• Drug: OCS-01, Oculis, Phase 2/3, Update (2023), (2022), NCT05066997  
• Drug: RG7774 (CANBERRA), Roche, Phase 2, Discontinued (2023), NCT04265261
• Gene therapy: aflibercept (ADVM-022), Adverum Biotechnologies, Phase 2 (INFINITY), Completed and Discontinued, NCT04418427
• Gene therapy: RGX-314 (ALTITUDE), AbbVie, Phase 2, Update (2023), NCT04567550
• Gene Therapy: 4D-150 (SPECTRA), 4D Molecular Therapeutics, Phase 1/2, Update (2023), NCT05930561

LEBER CONGENITAL AMAUROSIS (LCA)

• Gene therapy: LCA10, CEP290 c.2991+1655A>G in intron 26 (EDIT-101), Editas, Phase 1/2, Update  (2024), (2022), NCT03872479
• Gene therapy: RPE65, HG004 (STAR – US), (LIGHT – China), HuidaGene Therapeutics, Phase 1/2, Update (2024), NCT05906953, NCT06088992
• Gene Therapy: CEP290 (OCU400), Ocugen, Phase 1/2, Update (2024), (2023), NCT05203939
• Gene therapy: CEP290 (QR-110/Sepofarsen), ProQR Therapeutics (ILLUMINATE, BRIGHTEN), Phase 2/3, Canadian trial sites, Update (2022), Paused (2023), NCT03913143, NCT04855045
• Gene therapy: LCA1, GUCY2D (SAR439483), Atsena Therapeutics, Phase 1/2, Update (2023), (2022), NCT03920007
• Gene Therapy: LCA5 (AAV8.hLCA5), Opus Genetics, Phase 1/2, Update (2024), (2023), NCT05616793

LEBER HEREDITARY OPTIC NEUROPATHY (LHON)

• Gene Therapy: Mutation G11778A in mitochondrial DNA (cAAV2-P1ND4v2), Byron Lam/NEI, Phase 1, Update (2024), (2022), NCT02161380
• Gene Therapy: Mutation G11778A in mitochondrial DNA (Lumevoq, rAAV2/2-ND4/GS010), GenSight Biologics (REFLECT), Phase 3, Update (2024), (2023), NCT03293524
• Gene Therapy: ND1, NFS-02, Neurophth Therapeutics, Phase 1/2, Update (2023), NCT05820152
• Gene Therapy: ND4, NR082, Neurophth Therapeutics, Phase 1/2, Update (2024), NCT05293626

RETINITIS PIGMENTOSA (RP)

• Cell Therapy: neural progenitor cells, CNS10-NPC, Cedars-Sinai Medical Center, Phase 1, NCT04284293
• Cell Therapy: retinal progenitor cells, Centre d’Etude des Cellules Souches, Phase 1/2, Update (2023), NCT03963154
• Cell Therapy: retinal progenitor cells, jCyte, Phase 2, Completed, Update (2024), NCT03073733
• Cell Therapy: retinal progenitor cells, ReNeuron, Phase 1/2, Paused, NCT02464436
• Drug: Gene Agnostic, EA-2353, Endogena, Phase 1/2, Update (2023), NCT05392751  
• Drug: Gene agnostic, N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial sites, Phase 3, Update (2024), (2022), NCT05537220
• Drug: Rho-folding mutations, ADX-2191, Aldeyra Therapeutics, Phase 2, Completed, Update (2023), NCT05392179
• Drug: Disulfiram, University of Washington, Phase 1/2, NCT05626920
• Drug: Disulfiram, University of Rochester, Phase 1, NCT06319872
• Gene Therapy: Gene-agnostic (SPVN06), SparingVision, Phase1/2 (PRODYGY), Update (2024), (2023), NCT05748873
• Gene Therapy: OCU400 (liMeliGhT), Ocugen, Canadian trial sites (to be announced), Phase 3, Update (2024), NCT06388200
• Gene Therapy: NR2E3 and RHO (OCU400), Ocugen, Phase 1/2, Update (2024), (2023), NCT05203939 
• Gene therapy: PDE6B, Coave Therapeutics, Phase 1/2 Update (2024), (2023) , NCT03328130
• Gene therapy: PDE6A, STZ eye trial (PIGMENT), Phase 1/2, Update (2023), NCT04611503
• Gene therapy: RLBP1 (CPK850), Novartis, Phase 1/2, NCT03374657
• Gene therapy: USH2A exon13, QR-421a, ProQR Therapeutics, Phase 1/2 (STELLAR), Canadian trial site, Completed, (2023), NCT03780257
• Gene Therapy: CNGA1 (VG901), ViGeneron GmbH, Phase 1, NCT06291935
• RNA Therapy: RP11, PRPF31, PYC Therapeutics (Platypus), Phase 1/2, Update (2024), NCT05902962
• Optogenetic: RST-001, AbbVie, Phase 1/2, NCT02556736
• Optogenetic: BS01, Bionic Sight, Phase 1/2, Update (2023), (2021), NCT04278131
• Optogenetic: GS030, GenSight Biologics (PIONEER), Phase 1/2, Update (2023) NCT03326336
• Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, Update (2024), (2023) NCT05282953
• Optogenetic: vMCO-010, Nanoscope (RESTORE), Phase 2, Update (2024), (2023), NCT04945772
• RNA therapy: RHO P23H (QR-1123), ProQR Therapeutics (AURORA), Phase 1/2, Paused (2023), NCT04123626

STARGARDT DISEASE

• Drug: ABCA4, (ALK-001), (TEASE), Alkeus Pharmaceuticals , Phase 2, Update (2024), (2022), NCT02402660 
• Drug: Eximustat, Kubota Vision (SeaSTAR), Phase 3, Completed, Canadian Trial Sites, NCT03772665 
• Drug: STG-001, Stargazer Pharmaceuticals, Phase 2, Completed, NCT04489511
• Drug: Metformin, National Eye Institute, Phase 1/2, NCT04545736 
• Drug: Tinlarebant (STGD1), Belite Bio (DRAGON), Phase 3, Update (2024), (2023), NCT05244304, (DRAGON II), Phase 2/3, NCT06388083
• Drug: Zimura (Complement C5 inhibitor), Astellas, Phase 2, NCT03364153
• Gene therapy: OCU410ST, Ocugen (GARDian), Phase 1/2, Update (2024), NCT05956626
• Optogenetic: Multi-Characteristic Opsin (vMCO-010), Nanoscope Therapeutics (STARLIGHT), Phase 2, Completed. NCT05417126
• RNA editing therapy: ABCA4, ACDN-01 (STELLAR), Ascidian Therapeutics, Phase 1/2, Update (2024), NCT06467344

USHER SYNDROME

• Drug: N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial site, Phase 3, Update (2022), NCT05537220
• Drug: Ultevursen (QR-421a), USH2A exon13, ProQR Therapeutics (Sirius, Stellar), Phase 2/3, , Terminated, NCT05158296, NCT03780257
• Gene therapy: USH1B MYO7A, SAR421869, Sanofi, Phase 1/2, Terminated,  Update (2024), NCT01505062
• Drug: NPI-001, Nacuity Pharmaceuticals (SLO RP), Phase 1/2, Update (2023), NCT04355689

X-LINKED RETINITIS PIGMENTOSA (XLRP)

• Gene therapy: RPGR, AGTC-501 (SKYLINE), Beacon, Phase 2, Update (2024), NCT06333249
• Gene therapy: RPGR, AGTC-501 (VISTA), Beacon, Phase 2/3, Update (2024) NCT04850118
• Gene therapy: RPGR, AAV2/5, Meira GTx UK, Completed, NCT03252847
• Gene therapy: RPGR, AAV5-RPGR, Janssen Research and Development, Phase 3, Canadian Trial Site, Update (2022). NCT04671433
• Gene therapy: RPGR, BIIB112, NightstaRx/Biogen (XIRIUS), Phase 1/2, Completed, NCT03116113
• Gene therapy: RPGR, 4D-125, 4D Molecular Therapeutics, Phase 1/2, Update (2022) NCT04517149
• Gene Therapy: RPGR, FT-002, Frontera Therapeutics, Early Phase 1, NCT05874310

X-LINKED RETINOSCHISIS

• Gene therapy: RS1 (ATSN-201 Injection), Atsena Therapeutics (LIGHTHOUSE), Phase 1/2, Update (2024), NCT05878860
• Gene therapy: RS1, (Beacon Therapeutics), Phase 1/2, Completed, NCT02416622
• Gene therapy: RS1, National Eye Institute (NEI), Phase 1/2. Update (2022), NCT02317887

---

If you have questions about your eye health, contact our Health Information Line at healthinfo@fightingblindness.ca or 1.888.626.2995.

This page was last updated on August 27, 2024.