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RECRUITING

NCT06659445

PHASE2

Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD

Sponsor: ONL Therapeutics

View on ClinicalTrials.gov

Summary

The purpose of this study is to collect efficacy and safety information of intravitreal injection for 3 experimental arms, including 2 dose levels of ONL1204 and 2 treatment frequencies in patients with GA associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability and legal blindness globally. Associated with aging, cigarette smoking, obesity, diets low in certain nutrients, a lifestyle related to cardiac risk, and a growing list of genetic factors, AMD is becoming an increasingly prevalent public health concern, especially as the global population ages. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death that has demonstrated protection of multiple retinal cell types in numerous preclinical models of retinal disease, including models of dry AMD.

Official title: A Phase 2 Multicenter, Randomized, Double-masked, Sham-controlled, Reference-arm Study to Evaluate Efficacy and Safety of ONL1204 in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD)

Key Details

Gender

All

Age Range

55 Years - Any

Study Type

INTERVENTIONAL

Enrollment

324

Start Date

2025-10-28

Completion Date

2028-11

Last Updated

2026-08-26

Healthy Volunteers

Conditions

Geographic Atrophy (GA)Age - Related Macular Degeneration (AMD)

Interventions

DRUG

ONL1204 Opthalmic solution

Liquid formulation ONL1204 Ophthalmic Solution administered by intravitreal (IVT) injection

DRUG

Avacincaptad Pegol intravitreal solution

Liquid formulation Avacincaptad Pegol administered by intravitreal (IVT) injection

OTHER

Sham

Sham injection

Inclusion Criteria: 1. 55 years of age or older at Screening. 2. Able and willing to give informed consent and attend study visits. 3. Women or intersex individuals must be willing to use 2 forms of effective contraception during the study and for at least 90 days following the last dose of study drug, be postmenopausal for at least 12 months prior to study entry, or surgically sterile. If of childbearing potential, a negative urine pregnancy test is required at Screening and prior to the administration of study drug at each visit. 4. Men or intersex individuals with partners of childbearing potential must be willing to use permissible methods of contraception and refrain from sperm donation during the study and for at least 90 days following the last dose of study drug. 5. If currently using Age-related Eye Disease Study 2 (AREDS, AREDS2, or similar nutraceutical therapy at Screening, patient must be willing to continue use for the duration of the study. If not currently using AREDS2 or similar, patient must be willing to continue not to use therapy for the duration of the study. Patient must agree to choose either approach. Study Eye Inclusion Criteria: 6. BCVA of 24 letters or more using ETDRS charts in the study eye at Screening. 7. Intraocular pressure (IOP) of 22 mmHg or lower in the study eye at Screening. The GA lesion must meet all the following criteria: 8. Non-foveal GA associated with AMD without eMNV in the study eye per Investigator's evaluation and as confirmed by the central RC. 9. GA ≥0.85 disc area (DA) (2.125 mm2) and ≤8.05 DA (20.125 mm2) in the study eye as confirmed by the central RC. 10. If GA in the study eye is multifocal, at least 1 focal lesion must be ≥0.425 DA (1.063 mm2) as confirmed by the central RC. 11. Non-foveal GA (GA lesion up to the fovea, but not subfoveal) with the lesion's closest border 1725 microns or less from the foveal center in the study eye, as confirmed by the central RC. 12. The entire GA lesion in the study eye must be completely visualized on the macula-centered FAF image, must be able to be imaged in its entirety, and must not be contiguous with any areas of peripapillary atrophy per the Investigator's evaluation and as confirmed by the central RC. 13. A pattern of hyper-autofluorescence in the junctional zone of GA in the study eye must be present as confirmed by the central RC. Fellow Eye Inclusion Criterion: 14. BCVA of 19 letters or more using ETDRS charts in the fellow eye at Screening. Exclusion Criteria: 1. Current or planned participation in another investigational clinical study or use of any other investigational drugs or devices at least 6 months prior to enrollment or during the study period without prior written Sponsor approval. 2. Previous ophthalmic disease gene therapy or planned participation in any gene therapy clinical study during the study period. 3. Current or planned use of systemic complement inhibitors during the study period. 4. Any ocular or systemic condition that, in the opinion of the Investigator, makes the patient unsuitable for treatment with an investigational drug or that would compromise the safety or efficacy assessments of the study. 5. Treatment with any ocular or systemic medication that is known to be toxic to the lens, retina, or optic nerve (including, but not limited to, aminoglycosides, vancomycin, hydroxychloroquine, interferon, tacrolimus, cisplatin, bis-chloroethyl nitrosourea, carmustine, ethambutol, and tamoxifen) within 90 days prior to Screening or anticipated during the study period. 6. Any previous treatment of pentosan polysulfate (Elmiron®) is exclusionary. 7. Known allergy to fluorescein, povidone iodine, or any ingredients of the study drug or avacincaptad pegol intravitreal solution. 8. Individuals who are currently pregnant, planning to become pregnant, or are nursing at Screening or during the study period. Study Eye Exclusion Criteria: 9. Active ocular or periocular infection in the study eye. 10. Any contraindication to an IVT injection in the study eye. 11. Any media opacity in the study eye that limits visual acuity, clinical visualization of the retina, or retinal imaging as determined by the Investigator. 12. Previous IVT pharmaceutical treatment in the study eye with any agent except Food and Drug Administration (FDA)-approved complement inhibitors and treatment with FDA-approved complement inhibitors within 12 weeks prior to Screening. 13. Any history of incisional retinal surgery in the study eye including but not limited to scleral buckle or vitrectomy. (Retinal laser for a tear or hole 3 months or more prior to Screening is not exclusionary). 14. History of incisional glaucoma surgery including trabeculectomy, tube shunt, or minimally invasive glaucoma surgery in the study eye. (Glaucoma laser procedures performed at least 6 months prior to Screening are not exclusionary). 15. Yttrium aluminum garnet capsulotomy in the study eye within 1 month prior to Screening. 16. Cataract surgery in the study eye planned or expected during the study period. 17. Aphakia with absence of the posterior capsule in the study eye. (Pseudophakia with an open posterior capsule is not exclusionary). 18. Any current evidence or history in the study eye of exudative ("wet") AMD, including any RPE rips or evidence of actively leaking neovascularization anywhere in the retina based on the FA or SD-OCT as assessed per the Investigator's evaluation and confirmed by the central RC. 19. Any clinically significant retinal disease other than AMD except benign degenerative conditions (eg, Cobblestone degeneration) in the study eye. 20. Intraocular surgery in the study eye (including lens replacement surgery) within 3 months prior to Screening. See Exclusion Criteria 12 and 13 pertaining to retinal and glaucoma surgery, respectively. 21. Previous therapeutic radiation (including transpupillary thermotherapy, photodynamic therapy, or external-beam) in the region of the study eye. Fellow Eye Exclusion Criterion: 22. Planned or expected IVT injection in the fellow eye for treatment of GA with agents other than FDA-approved complement inhibitors (pegcetacoplan or avacincaptad pegol IVT solution \[Izervay™\]) during the study period. Either Eye Exclusion Criteria: 23. Active intraocular inflammation in either eye or a history of uveitis or endophthalmitis in either eye. 24. GA in either eye due to causes other than AMD. 25. Any ophthalmic condition in either eye that is likely to require surgery during the study period.

Locations (117)

Associated Retina Consultants

Gilbert, Arizona, United States

Associated Retina Consultants

Phoenix, Arizona, United States

California Retina Consultants

Bakersfield, California, United States

Retina-Vitreous Associates Medical Group

Beverly Hills, California, United States

Macula and Retina Institute

Glendale, California, United States

Salehi Retina Institute, dba Retina Associates of Southern California

Huntington Beach, California, United States

Desert Retina Consultants

Palm Desert, California, United States

Retinal Consultants Medical Group

Sacramento, California, United States

Bay Area Retina Associates

Walnut Creek, California, United States

Retina Consultants of Southern Colorado

Colorado Springs, Colorado, United States

Retina Group of New England

Waterford, Connecticut, United States

Florida Retina Institute

Orlando, Florida, United States

Retina Specialty Institute

Pensacola, Florida, United States

Retina Group of Florida

Sarasota, Florida, United States

Retina Associates of Florida, LLC

Tampa, Florida, United States

Northwestern Medical Group

Chicago, Illinois, United States

The University of Chicago Medicine Duchossois Center for Advanced Medicine

Chicago, Illinois, United States

Retina Associates, Ltd.

Elmhurst, Illinois, United States

Illinois Retina Associates, S.C.

Oak Park, Illinois, United States

Retina Partners Midwest, P.C.

Carmel, Indiana, United States

Retina Specialists

Towson, Maryland, United States

Retina Research Institute at New England Retina Consultants

Springfield, Massachusetts, United States

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, United States

Associated Retinal Consultants, P.C.

Royal Oak, Michigan, United States

Associated Retinal Consultants, P.C.

Traverse City, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

The Retina Institute

St Louis, Missouri, United States

Sierra Eye Associates

Reno, Nevada, United States

Retina Vitreous Surgeons of CNY, PC

Liverpool, New York, United States

Ophthalmic Consultants of Long Island

Oceanside, New York, United States

Retina Associates of Western NY

Rochester, New York, United States

Long Island Vitreoretinal Consultants

Westbury, New York, United States

North Carolina Retina Associates

Cary, North Carolina, United States

Duke Eye Center

Durham, North Carolina, United States

North Carolina Retina Associates

Greenville, North Carolina, United States

North Carolina Retina Associates, S.C.

Wake Forest, North Carolina, United States

Cleveland Clinic Foundation-Cole Eye Institute

Cleveland, Ohio, United States

Erie Retina Research, LLC (Clinic)

Erie, Pennsylvania, United States

Charleston Neuroscience Institute

Mt. Pleasant, South Carolina, United States

Palmetto Retina Center

West Columbia, South Carolina, United States

Vanderbilt Eye Institute

Nashville, Tennessee, United States

Austin Retina Associates

Austin, Texas, United States

Retina Consultants of Texas

Beaumont, Texas, United States

Retina Consultants of Texas

Bellaire, Texas, United States

Retina Consultants of Texas

Katy, Texas, United States

Valley Retina Institute, P.A.

McAllen, Texas, United States

Retina Associates of South Texas, PA

San Antonio, Texas, United States

Retina Consultants of Texas

San Antonio, Texas, United States

Retina Center of Texas

Southlake, Texas, United States

Retina Consultants of Texas

The Woodlands, Texas, United States

Piedmont Eye Center

Lynchburg, Virginia, United States

Pacific Northwest Retina

Bellevue, Washington, United States

Pacific Northwest Retina, PLLC

Silverdale, Washington, United States

Konventhospital der Barmherzigen Brüder Linz Abteilung für Augenheilkunde

Linz, Austria, Austria

Medizinische Universität Wien

Vienna, Austria, Austria

Vienna Institute for Research in Ocular Surgery - A Karl-Landsteiner-Institute

Vienna, Austria, Austria

St. Joseph's Health Care London - Ivey Eye Institute

London, Ontario, Canada

Toronto Retina Institute

Toronto, Ontario, Canada

Toronto Western Hospital, University Health Network

Toronto, Ontario, Canada

Clinique d'ophtalmologie des Laurentides

Boisbriand, Quebec, Canada

VISUS spol. s.r.o.

Náchod, Czech Republic, Czechia

Fakultni nemocnice Kralovske Vinohrady

Prague, Czech Republic, Czechia

VFN Praha

Prague, Czech Republic, Czechia

Axon Clinical, s.r.o.

Prague, Czech Republic, Czechia

NEMOS Medical Private Care s.r.o.

Sokolov, Czech Republic, Czechia

Augen-Zentrum-Nordwest

Ahaus, Germany, Germany

Universitäts-Augenklinik Bonn

Bonn, Germany, Germany

Internationale Innovative Ophthalmochirurgie GbR c/o MVZ Breyer Kaymak Klabe Augenchirurgie

Düsseldorf, Germany, Germany

Augenzentrum Prof. Dr. Koch GmbH

Frankfurt am Main, Germany, Germany

Philipps-Universität Marburg

Marburg, Germany, Germany

Uniklinikum Würzburg

Würzburg, Germany, Germany

General Hospital of Athens "G. Gennimatas", 1st University Eye Clinic

Athens, Greece, Greece

University General Hospital "Attikon", 2nd Department of Ophthalmology, University of Athens

Athens, Greece, Greece

University General Hospital of Heraklion

Heraklion, Greece, Greece

University General Hospital of Ioannina, Ophthalmology Clinica

Ioannina, Greece, Greece

University General Hospital of Patras, Department of Ophthalmology

Pátrai, Greece, Greece

Észak-Pesti Centrumkórház-Honvédkórház, Szemészeti Osztály

Budapest, Hungry, Hungary

Semmelweis Egyetem, Szemészeti Klinika Kutatóközpont

Budapest, Hungry, Hungary

Budapesti Bajcsy-Zsilinszky Kórház és Rendelőintézet, Szemészeti Osztály

Budapest, Hungry, Hungary

Budapest Retina Associates, Opthalmology

Budapest, Hungry, Hungary

Budapesti Jahn Ferenc Dél-pesti Kórház és Rendelőintézet, Szemészeti Osztály

Budapest, Hungry, Hungary

Debreceni Egyetem Klinikai Központ, Szemklinika

Debrecen, Hungry, Hungary

Pécsi Tudományegyetem Klinikai Kozpont, Szemeszeti Klinika

Pécs, Hungry, Hungary

Szegedi Tudományegyetem Szent-Györgyi Albert Klinikai Központ, Szemészeti Klinika

Szeged, Hungry, Hungary

Oculomedica Sp. z o.o.

Bydgoszcz, Poland, Poland

Optimum Profesorskie Centrum Okulistyki sp. z o.o.

Gdansk, Poland, Poland

Szpital Św. Róży sp. z o. o. sp. k.

Krakow, Poland, Poland

Centrum Medyczne Dietla 19 sp. z o.o.

Krakow, Poland, Poland

Klinika Okulistyczna Jasne Blonia sp. z o.o

Lodz, Poland, Poland

Bonifraterskie Centrum Medyczne sp. z o.o.

Lodz, Poland, Poland

Centrum Diagnostyki I Mikrochirurgii Oka Lens sp. Z o.o.

Olsztyn, Poland, Poland

AIBILI

Coimbra, Portugal, Portugal

Unidade Local de Saúde de Coimbra

Coimbra, Portugal, Portugal

Unidade Local de Saude de Gaia/Espinho

Gaia, Portugal, Portugal

Unidade Local De Saude De Santo Antonio

Porto, Portugal, Portugal

Centro de Oftalmología Barraquer

Barcelona, Spain, Spain

Institut Catalâ de Retina

Barcelona, Spain, Spain

Instituto Clinico Quirurgico de Oftalmologia (ICQO)

Bilbao, Spain, Spain

Hospital Arruzafa

Córdoba, Spain, Spain

Hospital Universitario Puerta de Hierro Majadahonda

Madrid, Spain, Spain

Hospital Universitario Virgen Macarena

Seville, Spain, Spain

Fundación de Oftalmologia Medica de la Comunitat Valenciana (FOM)

Valencia, Spain, Spain

Hospital Universitario Miguel Servet

Zaragoza, Spain, Spain

Augenarzt Praxisgemeinschaft Gutblick AG

Bern, Switzerland, Switzerland

Vista Augenklinik Binningen

Binningen, Switzerland, Switzerland

Hospital Ophthalmic Jules-Gonin Foundation

Lausanne, Switzerland, Switzerland

Swiss Visio Retina Research Center

Lausanne, Switzerland, Switzerland

Stadtspital Zurich Triemli, Augenklinik, Forschungszentrum, 2K.14

Zurich, Switzerland, Switzerland

Augenarzt Praxisgemeinschaft Gutblick AG

Zurich, Switzerland, Switzerland

Gloucestershire Hospitals NHS Foundation Trust

Gloucester, England, United Kingdom

King's College Hospital NHS Foundation Trust

London, England, United Kingdom

The Retina Clinic

London, England, United Kingdom

Newcastle Upon Tyne NHS Foundation Trust

Newcastle, England, United Kingdom

University Hospital Southampton NHS Foundation Trust

Southampton, England, United Kingdom

Sunderland Eye Infirmary

Sunderland, England, United Kingdom

Hull University Teaching Hospitals NHS Trust, Hull Royal Infirmary

York, England, United Kingdom

Royal Victoria Hospital (Ophthalmology Research Suite, EODU)

Belfast, Ireland, United Kingdom

Clinical Research Directory

Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (117)