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)
- 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 may not be approved by the FDA, meet basic safety standards or be appropriate for you. 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)
Gene therapy: CNGA3 (AGTC-402), Beacon Therapeutics, Phase 1/2, NCT02935517 - Gene therapy: CNGB3 (AGTC-401), Beacon Therapeutics, Phase 1/2, Discontinued (2023)
Gene therapy: CNGB3 (AGTC-401), Beacon Therapeutics, Phase 1/2, NCT02599922 - Gene therapy: CNGA3, STZ eye trial (COLOURBRIDGE), Phase 1/2, Update (2021)
Gene therapy: CNGA3, STZ eye trial (COLOURBRIDGE), Phase 1/2, 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)
Drug: AXT107 (DISCOVER). AsclepiX Therapeutics, Phase 1/2, NCT05859776 - Gene therapy: anti-VEGF (RGX-314) AbbVie: Phase 2 (AAVIATE), Update (2024)
Gene therapy: anti-VEGF (RGX-314) AbbVie: Phase 2 (AAVIATE), Update (2022)
Gene therapy: anti-VEGF (RGX-314) AbbVie: Phase 2 (AAVIATE), 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)
Gene Therapy: anti-VEGF (ADVM-022, Ixo-vec), Adverum Biotechnologies (LUNA), Phase 2, Update (2023)
Gene Therapy: anti-VEGF (ADVM-022, Ixo-vec), Adverum Biotechnologies (LUNA), Phase 2, NCT05536973 - Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1, Update (2022), Completed
Gene therapy: sCD59 (AAVCAGsCD59), Janssen, Phase 1,Completed, NCT03585556 - Gene Therapy: anti-VEGF and VEGF-C RNAi, 4D-150, 4D Molecular Therapeutics, Phase 1/2, Update (2024)
Gene Therapy: anti-VEGF and VEGF-C RNAi, 4D-150, 4D Molecular Therapeutics, Phase 1/2, Update (2022)
Gene Therapy: anti-VEGF and VEGF-C RNAi, 4D-150, 4D Molecular Therapeutics, Phase 1/2, NCT05197270
Geographic atrophy (Advanced Dry AMD)
- Bionics: PRIMA System, Pixium Vision, Update (2022)
Bionics: PRIMA System, Pixium Vision, 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)
Cell therapy : RPE (CPCB-RPE1), Regenerative Patch Technologies, Phase 1/2, 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)
Drug: Tinlarebant (PHOENIX), Belite Bio, Phase 3, Update, NCT05949593 - Drug: Zimura (Complement C5 inhibitor), IVERIC bio (GATHER1), Phase 3, Complete (2023). Acquired by Astellas and approved by FDA 2023.
Drug: Zimura (Complement C5 inhibitor), IVERIC bio (GATHER1), Phase 3, 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)
Gene therapy: GT005 (HORIZON, EXPLORE), Gyroscope/Novartis, Phase 2, NCT04566445
Gene therapy: GT005 (HORIZON, EXPLORE), Gyroscope/Novartis, Phase 2, NCT04437368 - Photobiomodulation: LumiThera, Update (2024)
Photobiomodulation: LumiThera, Update (2022)
Photobiomodulation: LumiThera, NCT04065490
CHOROIDEREMIA
- Gene therapy: REP1 (4D-110), 4D Molecular Therapeutics, Phase 1, Update (2023)
Gene therapy: REP1 (4D-110), 4D Molecular Therapeutics, Phase 1, NCT04483440 - Gene therapy: REP1 (BIIB111), NightstaRx/Biogen (STAR), Phase 3, Canadian trial sites, Completed
Gene therapy: REP1 (BIIB111), NightstaRx/Biogen (STAR), Phase 3, Canadian trial sites, NCT03496012 - Gene therapy: REP1 (SPK-7001), Spark Therapeutics, Phase 1/2, Completed
Gene therapy: REP1 (SPK-7001), Spark Therapeutics, Phase 1/2, NCT02341807 - Gene therapy: REP1, STZ eye trial (THOR), Phase 2, Completed
Gene therapy: REP1, STZ eye trial (THOR), Phase 2, NCT02671539 - Gene therapy: REP1, University of Oxford (REGENERATE), Phase 2, Completed
Gene therapy: REP1, University of Oxford (REGENERATE), Phase 2, NCT02407678 - Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, Update (2024)
Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, Update (2023)
Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, NCT05282953 (CHOROIDEREMIA)
Diabetes-related Eye Conditions
- Drug: EXN407, Exonate Limited, Phase 1/2, Update (2023)
Drug: EXN407, Exonate Limited, Phase 1/2, NCT04565756 - Drug: OCS-01, Oculis, Phase 2/3, Update (2023)
Drug: OCS-01, Oculis, Phase 2/3, Update (2022)
Drug: OCS-01, Oculis, Phase 2/3, NCT05066997 - Drug: RG7774 (CANBERRA), Roche, Phase 2, Discontinued (2023)
Drug: RG7774 (CANBERRA), Roche, Phase 2, NCT04265261 - Gene therapy: aflibercept (ADVM-022), Adverum Biotechnologies, Phase 2 (INFINITY), Completed and Discontinued
Gene therapy: aflibercept (ADVM-022), Adverum Biotechnologies, Phase 2 (INFINITY), NCT04418427 - Gene therapy: RGX-314 (ALTITUDE), AbbVie, Phase 2, Update (2023)
Gene therapy: RGX-314 (ALTITUDE), AbbVie, Phase 2, NCT04567550 - Gene Therapy: 4D-150 (SPECTRA), 4D Molecular Therapeutics, Phase 1/2, Update (2023)
Gene Therapy: 4D-150 (SPECTRA), 4D Molecular Therapeutics, Phase 1/2, NCT05930561
LEBER CONGENITAL AMAUROSIS (LCA)
- Gene therapy: LCA10, CEP290 c.2991+1655A>G in intron 26 (EDIT-101), Editas, Phase 1/2, Update (2024)
Gene therapy: LCA10, CEP290 c.2991+1655A>G in intron 26 (EDIT-101), Editas, Phase 1/2, Update (2022)
Gene therapy: LCA10, CEP290 c.2991+1655A>G in intron 26 (EDIT-101), Editas, Phase 1/2, NCT03872479 - Gene therapy: RPE65, HG004 (STAR – US), (LIGHT – China), HuidaGene Therapeutics, Phase 1/2, Update (2024)
Gene therapy: RPE65, HG004 (STAR – US), (LIGHT – China), HuidaGene Therapeutics, Phase 1/2, NCT05906953
Gene therapy: RPE65, HG004 (STAR – US), (LIGHT – China), HuidaGene Therapeutics, Phase 1/2, NCT06088992 - Gene Therapy: CEP290 (OCU400), Ocugen, Phase 1/2, Update (2024)
Gene Therapy: CEP290 (OCU400), Ocugen, Phase 1/2, Update (2023)
Gene Therapy: CEP290 (OCU400), Ocugen, Phase 1/2, NCT05203939 - Gene therapy: CEP290 (QR-110/Sepofarsen), ProQR Therapeutics (ILLUMINATE, BRIGHTEN), Phase 2/3, Canadian trial sites, Update (2022)
Gene therapy: CEP290 (QR-110/Sepofarsen), ProQR Therapeutics (ILLUMINATE, BRIGHTEN), Phase 2/3, Canadian trial sites, Update Paused
Gene therapy: CEP290 (QR-110/Sepofarsen), ProQR Therapeutics (ILLUMINATE, BRIGHTEN), Phase 2/3, Canadian trial sites, Update (2023)
Gene therapy: CEP290 (QR-110/Sepofarsen), ProQR Therapeutics (ILLUMINATE, BRIGHTEN), Phase 2/3, Canadian trial sites, NCT03913143
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)
Gene therapy: LCA1, GUCY2D (SAR439483), Atsena Therapeutics, Phase 1/2, Update (2022)
Gene therapy: LCA1, GUCY2D (SAR439483), Atsena Therapeutics, Phase 1/2, NCT03920007 - Gene Therapy: LCA5 (AAV8.hLCA5), Opus Genetics, Phase 1/2, Update (2024)
Gene Therapy: LCA5 (AAV8.hLCA5), Opus Genetics, Phase 1/2, Update (2023)
Gene Therapy: LCA5 (AAV8.hLCA5), Opus Genetics, Phase 1/2, NCT05616793
LEBER HEREDITARY OPTIC NEUROPATHY (LHON)
- Gene Therapy: Mutation G11778A in mitochondrial DNA (cAAV2-P1ND4v2), Byron Lam/NEI, Phase 1, Update (2024)
Gene Therapy: Mutation G11778A in mitochondrial DNA (cAAV2-P1ND4v2), Byron Lam/NEI, Phase 1, Update (2022)
Gene Therapy: Mutation G11778A in mitochondrial DNA (cAAV2-P1ND4v2), Byron Lam/NEI, Phase 1, NCT02161380 - Gene Therapy: Mutation G11778A in mitochondrial DNA (Lumevoq, rAAV2/2-ND4/GS010), GenSight Biologics (REFLECT), Phase 3, Update (2024)
Gene Therapy: Mutation G11778A in mitochondrial DNA (Lumevoq, rAAV2/2-ND4/GS010), GenSight Biologics (REFLECT), Phase 3, Update (2023)
Gene Therapy: Mutation G11778A in mitochondrial DNA (Lumevoq, rAAV2/2-ND4/GS010), GenSight Biologics (REFLECT), Phase 3, NCT03293524 - Gene Therapy: ND1, NFS-02, Neurophth Therapeutics, Phase 1/2, Update (2023)
Gene Therapy: ND1, NFS-02, Neurophth Therapeutics, Phase 1/2, NCT05820152 - Gene Therapy: ND4, NR082, Neurophth Therapeutics, Phase 1/2, Update (2024)
Gene Therapy: ND4, NR082, Neurophth Therapeutics, Phase 1/2, NCT05293626 (CHOROIDEREMIA)
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)
Cell Therapy: retinal progenitor cells, Centre d’Etude des Cellules Souches, Phase 1/2, NCT03963154 - Cell Therapy: retinal progenitor cells, jCyte, Phase 2, Completed, Update (2024)
Cell Therapy: retinal progenitor cells, jCyte, Phase 2, Completed, NCT03073733 - Cell Therapy: retinal progenitor cells, ReNeuron, Phase 1/2, Paused, NCT02464436
- Drug: Gene Agnostic, EA-2353, Endogena, Phase 1/2, Update (2023)
Drug: Gene Agnostic, EA-2353, Endogena, Phase 1/2, NCT05392751 - Drug: Gene agnostic, N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial sites, Phase 3, Update (2024)
Drug: Gene agnostic, N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial sites, Phase 3, Update (2022)
Drug: Gene agnostic, N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial sites, Phase 3, NCT05537220 - Drug: Rho-folding mutations, ADX-2191, Aldeyra Therapeutics, Phase 2, Completed, Update (2023)
Drug: Rho-folding mutations, ADX-2191, Aldeyra Therapeutics, Phase 2, Completed, 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)
Gene Therapy: Gene-agnostic (SPVN06), SparingVision, Phase1/2 (PRODYGY), Update (2023)
Gene Therapy: Gene-agnostic (SPVN06), SparingVision, Phase1/2 (PRODYGY), NCT05748873 - Gene Therapy: OCU400 (liMeliGhT), Ocugen, Canadian trial sites (to be announced), Phase 3, Update (2024)
Gene Therapy: OCU400 (liMeliGhT), Ocugen, Canadian trial sites (to be announced), Phase 3, NCT06388200 - Gene Therapy: NR2E3 and RHO (OCU400), Ocugen, Phase 1/2, Update (2024)
Gene Therapy: NR2E3 and RHO (OCU400), Ocugen, Phase 1/2, Update (2023)
Gene Therapy: NR2E3 and RHO (OCU400), Ocugen, Phase 1/2, NCT05203939 - Gene therapy: PDE6B, Coave Therapeutics, Phase 1/2 Update (2024)
Gene therapy: PDE6B, Coave Therapeutics, Phase 1/2 Update (2023)
Gene therapy: PDE6B, Coave Therapeutics, Phase 1/2, NCT03328130 - Gene therapy: PDE6A, STZ eye trial (PIGMENT), Phase 1/2, Update (2023)
Gene therapy: PDE6A, STZ eye trial (PIGMENT), Phase 1/2, 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
Gene therapy: USH2A exon13, QR-421a, ProQR Therapeutics, Phase 1/2 (STELLAR), Canadian trial site, (2023)
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)
RNA Therapy: RP11, PRPF31, PYC Therapeutics (Platypus), Phase 1/2, NCT05902962 - Optogenetic: RST-001, AbbVie, Phase 1/2, NCT02556736
- Optogenetic: BS01, Bionic Sight, Phase 1/2, Update (2023)
Optogenetic: BS01, Bionic Sight, Phase 1/2, Update (2021)
Optogenetic: BS01, Bionic Sight, Phase 1/2, NCT04278131 - Optogenetic: GS030, GenSight Biologics (PIONEER), Phase 1/2, Update (2023)
Optogenetic: GS030, GenSight Biologics (PIONEER), Phase 1/2, NCT03326336 - Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, Update (2024)
Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, Update (2023)
Optogenetic: KIO-301 (ABACUS), Kiora Pharmaceuticals, Phase 1/2, NCT05282953 - Optogenetic: vMCO-010, Nanoscope (RESTORE), Phase 2, Update (2024)
Optogenetic: vMCO-010, Nanoscope (RESTORE), Phase 2, Update (2023)
Optogenetic: vMCO-010, Nanoscope (RESTORE), Phase 2, NCT04945772 - RNA therapy: RHO P23H (QR-1123), ProQR Therapeutics (AURORA), Phase 1/2, Paused (2023)
RNA therapy: RHO P23H (QR-1123), ProQR Therapeutics (AURORA), Phase 1/2, NCT04123626
STARGARDT DISEASE
- Drug: ABCA4, (ALK-001), (TEASE), Alkeus Pharmaceuticals , Phase 2, Update (2024)
Drug: ABCA4, (ALK-001), (TEASE), Alkeus Pharmaceuticals , Phase 2, Update (2022)
Drug: ABCA4, (ALK-001), (TEASE), Alkeus Pharmaceuticals , Phase 2, Update (2024), (2022), NCT02402660 - Drug: Eximustat, Kubota Vision (SeaSTAR), Phase 3, Completed, Canadian Trial Sites
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)
Drug: Tinlarebant (STGD1), Belite Bio (DRAGON), Phase 3, Update (2023)
Drug: Tinlarebant (STGD1), Belite Bio (DRAGON), Phase 3, NCT05244304
Drug: Tinlarebant (STGD1), (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)
Gene therapy: OCU410ST, Ocugen (GARDian), Phase 1/2, NCT05956626 - Optogenetic: Multi-Characteristic Opsin (vMCO-010), Nanoscope Therapeutics (STARLIGHT), Phase 2, Completed.
Optogenetic: Multi-Characteristic Opsin (vMCO-010), Nanoscope Therapeutics (STARLIGHT), Phase 2. NCT05417126 - RNA editing therapy: ABCA4, ACDN-01 (STELLAR), Ascidian Therapeutics, Phase 1/2, Update (2024)
RNA editing therapy: ABCA4, ACDN-01 (STELLAR), Ascidian Therapeutics, Phase 1/2, NCT06467344
USHER SYNDROME
- Drug: N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial site, Phase 3, Update (2022)
Drug: N-acetylcysteine, Johns Hopkins University (NAC Attack), Canadian trial site, Phase 3, 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)
Gene therapy: USH1B MYO7A, SAR421869, Sanofi, Phase 1/2, Terminated, NCT01505062 - Drug: NPI-001, Nacuity Pharmaceuticals (SLO RP), Phase 1/2, Update (2023)
Drug: NPI-001, Nacuity Pharmaceuticals (SLO RP), Phase 1/2, NCT04355689
X-LINKED RETINITIS PIGMENTOSA (XLRP)
- Gene therapy: RPGR, AGTC-501 (SKYLINE), Beacon, Phase 2, Update (2024)
Gene therapy: RPGR, AGTC-501 (SKYLINE), Beacon, Phase 2, NCT06333249 - Gene therapy: RPGR, AGTC-501 (VISTA), Beacon, Phase 2/3, Update (2024)
Gene therapy: RPGR, AGTC-501 (VISTA), Beacon, Phase 2/3, NCT04850118 - Gene therapy: RPGR, AAV2/5, Meira GTx UK, Completed
Gene therapy: RPGR, AAV2/5, Meira GTx UK, NCT03252847 - Gene therapy: RPGR, AAV5-RPGR, Janssen Research and Development, Phase 3, Canadian Trial Site, Update (2022)
Gene therapy: RPGR, AAV5-RPGR, Janssen Research and Development, Phase 3, Canadian Trial Site, NCT04671433 - Gene therapy: RPGR, BIIB112, NightstaRx/Biogen (XIRIUS), Phase 1/2, Completed
Gene therapy: RPGR, BIIB112, NightstaRx/Biogen (XIRIUS), Phase 1/2, NCT03116113 - Gene therapy: RPGR, 4D-125, 4D Molecular Therapeutics, Phase 1/2, Update (2022)
Gene therapy: RPGR, 4D-125, 4D Molecular Therapeutics, Phase 1/2, 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)
Gene therapy: RS1 (ATSN-201 Injection), Atsena Therapeutics (LIGHTHOUSE), Phase 1/2, NCT05878860 - Gene therapy: RS1, (Beacon Therapeutics), Phase 1/2, Completed
Gene therapy: RS1, (Beacon Therapeutics), Phase 1/2, NCT02416622 - Gene therapy: RS1, National Eye Institute (NEI), Phase 1/2. Update (2022)
Gene therapy: RS1, National Eye Institute (NEI), Phase 1/2, 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.
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