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Clinical Trials

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 diabetic macular edema (DME).

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)
  • Trial Phase
  • Other information including if there is a Canadian trial site, or links to completed trial results or updates
  • number. This will link out to trial page on the website

You can find more information about these or other clinical trials at 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.



Neovascular (wet) AMD

  • Gene therapy: anti-VEGF (RGX-314), Regenxbio Inc (AAVIATE), Phase 2, NCT04514653
  • Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1, NCT03585556

Geographic atrophy (Advanced Dry AMD)

  • Drug: Zimura (Complement C5 inhibitor), IVERIC bio (GATHER1), Phase 3, Canadian trial sites, Update (2020), NCT04435366
  • Drug: Pegcetacoplan/APL-2 (Complement C3 inhibitor), Apellis (GALE), Phase 3, NCT04770545
  • Drug: Ionis-FB-LRx (Complement Factor B inhibitor), Ionis (GOLDEN), Phase 2, Canadian trial site, NCT03815825
  • Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1, NCT03585556
  • Gene therapy: GT005 (Complement factor), Gyroscope (FOCUS), Phase 1/2, Update (2021), NCT03846193
  • Stem cell: hESC-RPE (OpRegen), Lineage Cell Therapeutics, Phase 1/2, NCT02286089
  • Stem cell: iPSC-RPE/PLGA, NEI, Phase 1/2, NCT04339764
  • Stem cell: hESC-RPE (CPCB-RPE1), Regenerative Patch Technologies, Phase 1/2, Update (2021), NCT02590692.
  • Stem cell: hSC-RPE (ASP7317), Astellas, Phase 1, NCT03178149


  • Gene therapy: REP1 (BIIB111), NightstaRx/Biogen (STAR), Phase 3, Canadian trial sites, Completed, NCT03496012
  • Gene therapy: REP1 (SPK-7001), Spark Therapeutics, Phase 1/2, NCT02341807
  • Gene therapy: REP1, STZ eye trial (THOR), Phase 2, Completed, NCT02671539
  • Gene therapy: REP1, University of Oxford (REGENERATE), Phase 2, Completed, NCT02407678
  • Gene therapy: REP1 (4D-110), 4D Molecular Therapeutics, Phase 1, Update (2021), NCT04483440


  • Gene therapy: aflibercept (ADVM-022), Adverum Biotechnologies, Phase 2 (INFINITY), Completed, NCT04418427


  • Gene therapy: CEP290 c.2991+1655A>G in intron 26 (EDIT-101), Editas, Phase 1/2, NCT03872479
  • Gene therapy: GUCY2D (SAR439483), Atsena Therapeutics, Phase 1/2, Update (2021), NCT03920007
  • Gene therapy: RPE65 (OPTIRPE65), MeiraGTx, long term follow up of Completed Phase 1/2 trial, NCT02946879
  • Gene therapy (RNA): CEP290 (QR-110/Sepofarsen), ProQR Therapeutics (ILLUMINATE), Phase 2/3, Canada trial site, NCT03913143
  • Gene therapy (RNA): CEP290 (QR-110/Sepofarsen), ProQR Therapeutics, Phase 2/3 (BRIGHTEN), under 8 years old, NCT04855045


  • Gene Therapy: Mutation G11778A in mitochondrial DNA (cAAV2-P1ND4v2), Byron Lam/NEI, Phase 1, Update (2017), NCT02161380
  • Gene Therapy: Mutation G11778A in mitochondrial DNA (rAAV2/2-ND4/GS010), GenSight Biologics (REFLECT), Phase 3, NCT03293524
  • Gene Therapy: Mutation G11778A in mitochondrial DNA (rAAV2/2-ND4/GS010), GenSight Biologics (RESCUE), Phase 3, Completed, NCT02652767


  • Gene therapy: RS1, Applied Genetic Technologies Cord (AGTC), Phase 1/2, Completed, NCT02416622
  • Gene therapy: RS1, National Eye Institute (NEI), Phase 1/2. Update (2021), NCT02317887


  • Gene therapy: PDE6B, Horama, Phase 1/2, NCT03328130
  • Gene therapy: PDE6A, STZ eye trial (PIGMENT), Phase 1/2, NCT04611503
  • RNA therapy: RHO P23H, QR-1123, ProQR Therapeutics (AURORA), Phase 1/2, NCT04123626
  • Gene therapy: USH2A exon13, QR-421a, ProQR Therapeutics, Phase 1/2 (STELLAR), Canadian trial site, Update (2021), NCT03780257
  • Gene therapy: RLBP1, CPK850, Novartis, Phase 1/2, NCT03374657
  • Optogenetic: vMCO-010, Nanoscope (RESTORE), Phase 2, NCT04945772
  • Optogenetic: RST-001, Allergan, Phase 1/2, NCT02556736
  • Optogenetic: GS030, GenSight Biologics (PIONEER), Phase 1/2, Update (2021), NCT03326336
  • Optogenetic: BS01, Bionic Sight, Phase 1/2, Update (2021), NCT04278131
  • Stem cell: hRPC, jCyte, Phase 2, Completed, NCT03073733
  • Stem cell: hRPC, ReNeuron, Phase 1/2, Update (2019), NCT02464436
  • Stem cell: hESC-RPE Centre d’Etude des Cellules Souches, Phase 1/2, NCT03963154
  • Stem cell: hNPC, Cedars-Sinai Medical Center, Phase 1, NCT04284293


  • Drug: ABCA4, Alkeus Pharmaceuticals (TEASE), Phase 2, Update (2019), NCT02402660
  • Drug: Zimura (Complement C5 inhibitor), IVERIC bio, Phase 2, NCT03364153
  • Drug: Eximustat, Kubota Vision, Phase 2, NCT03033108
  • Drug: STG-001, Stargazer Pharmaceuticals, Phase 2, Completed, NCT04489511
  • Stem cell: hESC-RPE, ASP7317, Astellas Institute for Regenerative Medicine, Phase 1, long-term follow up study, NCT03178149.


  • Gene therapy: USH1B MYO7A, SAR421869, Sanofi, Phase 1/2, Terminated, NCT02065011
  • Gene therapy: USH2A exon13, QR-421a, ProQR Therapeutics (STELLAR), Phase 1/2, Canadian trial site, Update (2021), NCT03780257
  • Drug: NPI-001, Nacuity Pharmaceuticals (SLO RP), Phase 1/2, NCT04355689


  • Gene therapy: RPGR, AGTC-501, AGTC, Phase 2/3, NCT04850118
  • Gene therapy: RPGR, AAV2/5-RPGR, MeiraGTx, Phase 1/2, Update (2021), NCT03252847
  • Gene therapy: RPGR, AAV5-RPGR, MeiraGTx, Phase 3, 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 (2021), NCT04517149

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

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