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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 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)
  • Trial Phase
  • 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

AGE-RELATED MACULAR DEGENERATION (AMD)

Neovascular (wet) AMD

  • Gene therapy: anti-VEGF (RGX-314), Regenxbio Inc (AAVIATE), Phase 2, Update (2022), NCT04514653
  • Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1, Update (2022),  NCT03585556

Geographic atrophy (Advanced Dry AMD)

  • Bionics: PRIMA System, Pixium Vision, Update (2022), NCT04676854
  • Drug: Zimura (Complement C5 inhibitor), IVERIC bio (GATHER1), Phase 3, Canadian trial sites, Update (2022), NCT04435366
  • Drug: Pegcetacoplan/APL-2 (Complement C3 inhibitor), Apellis (GALE), Phase 3, Canadian trial sites, Update (2022NCT04770545
  • Drug: Ionis-FB-LRx (Complement Factor B inhibitor), Ionis (GOLDEN), Phase 2, Canadian trial sites, NCT03815825
  • Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1, Update (2022), NCT03144999  
  • Gene therapy: GT005 (Complement factor), Gyroscope (FOCUS), Phase 1/2, Update (2021), NCT03846193
  • Photobiomodulation: PBM (Valeda), LumiThera, Update (2022), NCT04065490  
  • Stem cell: hESC-RPE (OpRegen), Hoffmann-La Roche, Phase 1/2, Update (2020), CompletedNCT02286089
  • Stem cell: iPSC-RPE/PLGA, NEI, Phase 1/2, Update (2022), NCT04339764
  • Stem cell: hESC-RPE (CPCB-RPE1), Regenerative Patch Technologies, Phase 1/2, Update (2021), (2022), NCT02590692.
  • Stem cell: hSC-RPE (ASP7317), Astellas, Phase 1, NCT03178149

CHOROIDEREMIA

Diabetes-related Eye Conditions

LEBER CONGENITAL AMAUROSIS (LCA)

  • Gene therapy: CEP290 c.2991+1655A>G in intron 26 (EDIT-101), Editas, Phase 1/2, Update (2022), NCT03872479
  • Gene therapy: GUCY2D (SAR439483), Atsena Therapeutics, Phase 1/2, Update (2022), 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, Canadian trial sites, Update (2022), Paused,  NCT03913143
  • Gene therapy (RNA): CEP290 (QR-110/Sepofarsen), ProQR Therapeutics, Phase 2/3 (BRIGHTEN), under 8 years old, Canadian trial sites, Update (2022), Paused, NCT04855045

LEBER HEREDITARY OPTIC NEUROPATHY (LHON)

  • Gene Therapy: Mutation G11778A in mitochondrial DNA (cAAV2-P1ND4v2), Byron Lam/NEI, Phase 1, Update (2022), NCT02161380
  • Gene Therapy: Mutation G11778A in mitochondrial DNA (rAAV2/2-ND4/GS010), GenSight Biologics (REFLECT), Phase 3, Update (2021), NCT03293524
  • Gene Therapy: Mutation G11778A in mitochondrial DNA (rAAV2/2-ND4/GS010), GenSight Biologics (RESCUE), Phase 3, CompletedNCT02652767

RETINOSCHISIS

  • Gene therapy: RS1, Applied Genetic Technologies Cord (AGTC), Phase 1/2, CompletedNCT02416622
  • Gene therapy: RS1, National Eye Institute (NEI), Phase 1/2. Update (2021), NCT02317887

RETINITIS PIGMENTOSA (RP)

  • Drug: N-acetylcysteine, Johns Hopkins University, Canadian trial sites, Phase 3, Update (2022), NCT05537220  
  • Drug: KIO-301, Kiora Pharmaceuticals, Phase 1, Update (2022), NCT05282953  
  • Gene therapy: PDE6B, Coave Therapeutics, Phase 1/2, NCT03328130
  • Gene therapy: PDE6A, STZ eye trial (PIGMENT), Phase 1/2, NCT04611503
  • Gene therapy: OCU400, Ocugen, Phase 1/2, Update (2022), NCT05203939   
  • Gene therapy: USH2A exon13, QR-421a, ProQR Therapeutics, Phase 1/2 (STELLAR), Canadian trial site, Update (2021), (2022), PausedNCT03780257
  • Gene therapy: RLBP1, CPK850, Novartis, Phase 1/2, NCT03374657
  • RNA therapy: RHO P23H, QR-1123, ProQR Therapeutics (AURORA), Phase 1/2, Update (2019), Paused, NCT04123626
  • Optogenetic: vMCO-010, Nanoscope (RESTORE), Phase 2, Update (2022), NCT04945772
  • Optogenetic: RST-001, Allergan, Phase 1/2, Update (2022), NCT02556736
  • Optogenetic: GS030, GenSight Biologics (PIONEER), Phase 1/2, Update (2021), NCT03326336
  • Optogenetic: BS01, Bionic Sight, Phase 1/2, Update (2021), NCT04278131
  • Stem cell: EA-2353, Endogena, Phase 1/2, Update (2022), NCT05392751  
  • Stem cell: hRPC, jCyte, Phase 2, Completed, NCT03073733
  • Stem cell: hRPC, ReNeuron, Phase 1/2, Paused. Update (2021), 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

STARGARDT DISEASE

  • Drug: ABCA4, Alkeus Pharmaceuticals (TEASE), Phase 2, Update (2022), NCT02402660
  • Drug: Zimura (Complement C5 inhibitor), IVERIC bio, Phase 2, Update (2022NCT03364153
  • Drug: Eximustat, Kubota Vision, Phase 2, CompletedNCT03033108
  • Drug: Metformin, National Eye Institute, Phase 1/2, NCT04545736 
  • Drug: Tinlarebant, Belite Bio, Phase 3, Update (2022), NCT05244304
  • Drug: STG-001, Stargazer Pharmaceuticals, Phase 2, Completed, NCT04489511
  • Optogenetic: Multi-Characteristic Opsin (vMCO-010), Nanoscope Therapeutics, Phase 2, Update (2022), NCT05417126
  • Stem cell: hESC-RPE, ASP7317, Astellas Institute for Regenerative Medicine, Phase 1, long-term follow up studyNCT03178149

USHER SYNDROME

  • Gene therapy: USH1B MYO7A, SAR421869, Sanofi, Phase 1/2, TerminatedNCT02065011
  • Gene therapy: USH2A exon13, QR-421a, ProQR Therapeutics (STELLAR), Phase 1/2, Canadian trial site, Update (2021), (2022), PausedNCT03780257
  • Drug: NPI-001, Nacuity Pharmaceuticals (SLO RP), Phase 1/2, Update (2022), NCT04355689

X-LINKED RETINITIS PIGMENTOSA (XLRP)


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 November 21, 2022.

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