Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration
NCT ID: NCT03525600
Last Updated: 2023-06-18
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
621 participants
INTERVENTIONAL
2018-08-31
2022-06-20
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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APL-2 15mg 0.1 mL monthly for 24 months
A single dose of 15 mg APL-2/0.1 mL will be administered via intravitreal injection in this study. Subjects will receive an injection every month
APL-2
Complement (C3) Inhibitor
APL-2 15mg 0.1 mL EOM for 24 months
A single dose of 15 mg APL-2/0.1 mL will be administered via intravitreal injection in this study. Subjects will receive an injection every other month
APL-2
Complement (C3) Inhibitor
Sham Procedure Monthly for 24 months
Sham Procedure for 24 months
Sham Procedure
Subjects will receive a Sham procedure every month
Sham Procedure Every Other Month for 24 months
Sham Procedure every other month for 24 months
Sham Procedure
Subjects will receive a Sham procedure every other month
Interventions
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APL-2
Complement (C3) Inhibitor
APL-2
Complement (C3) Inhibitor
Sham Procedure
Subjects will receive a Sham procedure every month
Sham Procedure
Subjects will receive a Sham procedure every other month
Eligibility Criteria
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Inclusion Criteria
* Normal Luminance best corrected visual acuity of 24 letters or better using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately 20/320 Snellen equivalent).
* Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center.
* The GA lesion must meet the following criteria as determined by the central reading center's assessment of Fundus Autofluorescence (FAF) imaging at screening:
* Total GA area must be ≥ 2.5 and ≤ 17.5 mm2 (1 and 7 disk areas \[DA\] respectively)
* If GA is multifocal, at least one focal lesion must be ≥ 1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 4a.
* The entire GA lesion must be completely visualized on the macula centered image and must be able to be imaged in its entirety and not contiguous with any areas of peripapillary atrophy.
* Presence of any pattern of hyperautofluorescence in the junctional zone of GA. Absence of hyperautofluorescence (i.e. pattern = none) is exclusionary.
* Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the Investigator.
* Female subjects must be:
* Women of non-child-bearing potential (WONCBP), or
* Women of child-bearing potential (WOCBP) with a negative serum pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study and refrain from breastfeeding for the duration of the study.
* Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study.
* Willing and able to give informed consent and to comply with the study procedures and assessments.
Exclusion Criteria
* Spherical equivalent of the refractive error demonstrating \> 6 diopters of myopia or an axial length \>26 mm.
* Any history or active choroidal neovascularization (CNV), associated with AMD or any other cause, including any evidence of retinal pigment epithelium rips or evidence of neovascularization anywhere based on SD-OCT imaging and/or fluorescein angiography as assessed by the Reading Center.
* Presence of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function, including but not limited to, uveitis, other macular diseases (e.g. clinically significant epiretinal membrane (ERM), full thickness macular hole or uncontrolled glaucoma/ocular hypertension. Benign conditions in the opinion of the investigator such as peripheral retina dystrophy are not exclusionary).
* Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
* History of laser therapy in the macular region.
* Aphakia or absence of the posterior capsule. Note: YAG laser posterior capsulotomy for posterior capsule opacification done at least 60 days prior to screening is not exclusionary.
* Any ocular condition other than GA secondary to AMD that may require surgery or medical intervention during the study period or, in the opinion of the Investigator, could compromise visual function during the study period.
* Any contraindication to IVT injection including current ocular or periocular infection.
* History of prior intravitreal injection.
* Prior participation in another interventional clinical study for intravitreal therapies in either eye (including subjects receiving sham).
* Prior participation in another interventional clinical study for geographic atrophy in either eye including investigational oral medication and placebo.
* Participation in any systemic experimental treatment or any other systemic investigational new drug within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of study treatment. Note: clinical trials solely involving observation, over-the-counter vitamins, supplements, or diets are not exclusionary.
* Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the 24-month treatment period unlikely, or would make the subject an unsafe study candidate.
* Any screening laboratory value (hematology, serum chemistry or urinalysis) that in the opinion of the Investigator is clinically significant and not suitable for study participation.
* Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to APL-2 or any of the excipients in APL-2 solution.
60 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Retinal Research Institute
Phoenix, Arizona, United States
Associated Retina Consultants, Ltd
Phoenix, Arizona, United States
Retina Institute of California dba Acuity Eye Grp
Arcadia, California, United States
California Retina Consultants
Bakersfield, California, United States
Retina Vitreous Associates Medical Group
Beverly Hills, California, United States
The Retina Partners
Encino, California, United States
The Gavin Herbert Eye Institute/UC Irvine
Irvine, California, United States
Northern California Retina Vitreous Associates
Mountain View, California, United States
Retina Institute of California Medical Group
Palm Desert, California, United States
Byers Eye Institute at Standford, Stanford School of Medicine
Palo Alto, California, United States
Retina Consultants San Diego
Poway, California, United States
Retina Consultants of Southern California
Redlands, California, United States
California Retina Consultants
Santa Barbara, California, United States
Bay Area Retina Associates
Walnut Creek, California, United States
Danbury Eye Physicians & Surgeons, P.C. - Danbury
Danbury, Connecticut, United States
New England Retina Associates
Hamden, Connecticut, United States
Retina Group of New England,PC
Waterford, Connecticut, United States
Florida Eye Microsurgical Institute, Inc.
Boynton Beach, Florida, United States
Pinnacle Research Institute
Fort Lauderdale, Florida, United States
Retina Health Center
Fort Myers, Florida, United States
Bascom Palmer Eye Institute
Palm Beach Gardens, Florida, United States
Eye Associates of Pinellas
Pinellas Park, Florida, United States
East Florida Eye Institute
Stuart, Florida, United States
Southern Vitreoretinal Associates
Tallahassee, Florida, United States
Retina Associates of Florida
Tampa, Florida, United States
University of South Florida (USF) Eye Institute
Tampa, Florida, United States
Southeast Retina Center, PC
Augusta, Georgia, United States
Georgia Retina
Marietta, Georgia, United States
Midwest Eye Institute
Indianapolis, Indiana, United States
Sabates Eye Center
Leawood, Kansas, United States
Elman Retina Group, PA
Baltimore, Maryland, United States
Cumberland Valley Retina Consultants, PC
Hagerstown, Maryland, United States
Mid Atlantic Retina Specialists
Hagerstown, Maryland, United States
Retina Specialists
Towson, Maryland, United States
Ophthalmic Consultants of Boston
Boston, Massachusetts, United States
New England Retina Consultants, PC
Springfield, Massachusetts, United States
Associated Retinal Consultants, P.C
Grand Rapids, Michigan, United States
Retina Specialists of Michigan / Foundation for Vision
Grand Rapids, Michigan, United States
Retina Consultants of Michigan
Southfield, Michigan, United States
Associated Retinal Consultants PC
Traverse City, Michigan, United States
Sierra Eye Associates
Reno, Nevada, United States
Retina Associates of New Jersey (NJ Retina)
Teaneck, New Jersey, United States
Long Island Vitreoretinal Consultants
Great Neck, New York, United States
Vitreous Retina Macula Consultants of NY
New York, New York, United States
Western Carolina Retinal Associates
Asheville, North Carolina, United States
Charlotte Eye Ear Nose and Throat Associates, PS
Charlotte, North Carolina, United States
Graystone Eye
Hickory, North Carolina, United States
Retina Associates of Cleveland, Inc.
Cleveland, Ohio, United States
Retina Associates of Cleveland, Inc
Cleveland, Ohio, United States
Retina Associates of Cleveland
Cleveland, Ohio, United States
Cleveland Clinic, Cole Eye Institute
Cleveland, Ohio, United States
The Ohio State University
Columbus, Ohio, United States
Retina Associates of Cleveland, Inc.
Youngstown, Ohio, United States
Retina Northwest, PC
Portland, Oregon, United States
Eye Health Northwest
Portland, Oregon, United States
Mid Atlantic Retina
Philadelphia, Pennsylvania, United States
Black Hills Regional Eye Institute
Rapid City, South Dakota, United States
Tennessee Retina, PC
Nashville, Tennessee, United States
Retina Research Institute of Texas
Abilene, Texas, United States
Southwest Retina Specialists
Amarillo, Texas, United States
Retina Consultants of Austin (Retina Research Center)
Austin, Texas, United States
Retina Consultants of Houston, PA
Houston, Texas, United States
Valley Retina Institute, PA
McAllen, Texas, United States
Medical Center Ophthalmology Associates
San Antonio, Texas, United States
Retinal Consultants of San Antonio
San Antonio, Texas, United States
Retina Consultants of Houston
The Woodlands, Texas, United States
Retina Associates of Utah, PC
Murray, Utah, United States
University of Virginia
Charlottesville, Virginia, United States
The Retina Group of Washington
Fairfax, Virginia, United States
Virginia Retina Center
Warrenton, Virginia, United States
Vitreoretinal Associates of Washington
Bellevue, Washington, United States
Spokane Eye Clinical Research
Spokane, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
Fundacion Zambrano
Buenos Aires, Buenos Aires F.D., Argentina
Microcirugia Ocular
El Rosario, Corrientes Province, Argentina
Grupo Laser Vision
Rosario, Santa Fe Province, Argentina
Organizacion Medica de investigacion
Buenos Aires, , Argentina
Centro Oftalmologico Dr Charles
Buenos Aires, , Argentina
Diagnostico Ocular
Buenos Aires, , Argentina
Centro Privado de Ojos Romagosa SA
Córdoba, , Argentina
Instituto Oftalmologico de Cordoba
Córdoba, , Argentina
Oftar Mendoza SRL
Mendoza, , Argentina
Oftalmologos Especialistas
Rosario, , Argentina
Sydney Retina
Sydney, New South Wales, Australia
Centre for Eye Research Australia
East Melbourne, Victoria, Australia
Retina and Eye Consultants
Hurstville, , Australia
Clinica Ocular Oftalmologia LTDA
Vitória, Espírito Santo, Brazil
Clinica Oftalmologica Sao Lucas
Osasco, São Paulo, Brazil
IPEPO - Instituto Da Visao
São Paulo, Vila Clementino, Brazil
Instituto da Visão - Hospital de Olhos Ltda
Belo Horizonte, , Brazil
Hospital De Clinicas De Porto Alegre
Porto Alegre, , Brazil
UNIFESP - Federal University
São Paulo, , Brazil
Ivey Eye Institute
London, Ontario, Canada
University of Ottawa Eye Institute
Ottawa, Ontario, Canada
DRY AMD Clinic - St. Michael's Hospital
Toronto, Ontario, Canada
Retina Centre of Ottawa
Ottawa, , Canada
Fakultní nemocnice Ostrava
Ostrava-Poruba, , Czechia
OFTEX Eye Clinic
Pardubice, , Czechia
AXON Clinical, S.R.O.
Prague, , Czechia
University Hospital Kralovske Vinochrady
Vinohrady, , Czechia
Centre Hospitalier Intercommunal de Créteil
Créteil, , France
Hopital de la Croix-Rousse
Lyon, , France
Centre Monticelli Paradis
Marseille, , France
CHU de Nantes - Hotel Dieu
Nantes, , France
Centre Ophtalmologique de l´Odéon
Paris, , France
Centre Ophthalmologique Saint-Exupery
Saint-Cyr-sur-Loire, , France
Maison Rouge Ophthalmologic Center
Strasbourg, , France
Universitäts-Augenklinik Bonn
Bonn, , Germany
University Hospital Cologne
Cologne, , Germany
Klinikum der Stadt Ludwigshafen gGmbH
Ludwigshafen, , Germany
Klinikum rechts der Isar
München, , Germany
Universitätsklinikum Regensburg
Regensburg, , Germany
University Hospital Würzburg
Würzburg, , Germany
Shamir Medical Center
Be’er Ya‘aqov, , Israel
Rambam Medical Center
Haifa, , Israel
Meir Medical Center
Kfar Saba, , Israel
Rabin Medical Center
Petah Tikva, , Israel
Kaplan Medical Center
Rehovot, , Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, , Israel
Ospedale San Raffaele
Milan, , Italy
Luigi Sacco Hospital
Milan, , Italy
Retina Specialist
Auckland, , New Zealand
Hamilton Eye Clinic
Hamilton, , New Zealand
Oftalmika Eye Hospital
Bydgoszcz, , Poland
Jasne Blonia Eye Clinic
Lodz, , Poland
Centrum Diagnostyki i Mikrochirurgii Oka - LENS
Olsztyn, , Poland
Centrum Medyczne UNO-MED
Tarnów, , Poland
Emanuelli Research and Development Center
Arecibo, , Puerto Rico
Centro Médico Teknon
Barcelona, , Spain
Instituto Oftalmologico Gómez-Ulla
Santiago de Compostela, , Spain
Hospital Universitario Rio Hortega
Valladolid, , Spain
Moorfields Eye Hospital NHS Foundation Trust
London, England, United Kingdom
Eye Clinic, Acre Mill Outpatients, Huddersfield Royal Infirmary
Huddersfield, West Yorkshire, United Kingdom
Bristol Eye Hospital
Bristol, , United Kingdom
St James's University Hospital
Leeds, , United Kingdom
Leicester Royal Infirmary
Leicester, , United Kingdom
London North West University Hospital Trust
London, , United Kingdom
King's College Hospital NHS Trust
London, , United Kingdom
Oxford Eye Hospital
Oxford, , United Kingdom
Salisbury NHS Foundation Trust
Salisbury, , United Kingdom
Sunderland Eye Infirmary
Sunderland, , United Kingdom
Countries
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References
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Sadda S, Hatcher KA, Shah BK, Kondapalli SS, Li C, Baumal CR. Outer Retinal Tubulation and Geographic Atrophy: A Natural History Analysis of Sham Observed Eyes from the OAKS and DERBY Trials. Ophthalmol Ther. 2025 Jul;14(7):1611-1619. doi: 10.1007/s40123-025-01156-5. Epub 2025 May 19.
Wykoff CC, Holz FG, Chiang A, Boyer D, Dhoot DS, Loewenstein A, Mones J, Heier J, Abbey AM, Singerman LJ, Vajzovic L, Lin J, Li C, Vilupuru A, Baumal CR; OAKS, DERBY, and GALE Investigators. Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE. Am J Ophthalmol. 2025 Aug;276:350-364. doi: 10.1016/j.ajo.2025.04.016. Epub 2025 Apr 23.
Fu DJ, Bagga P, Naik G, Glinton S, Faes L, Liefers B, Lima R, Wignall G, Keane PA, Ioannidou E, Ribeiro Reis AP, McKeown A, Scheibler L, Patel PJ, Moghul I, Pontikos N, Balaskas K. Pegcetacoplan Treatment and Consensus Features of Geographic Atrophy Over 24 Months. JAMA Ophthalmol. 2024 Jun 1;142(6):548-558. doi: 10.1001/jamaophthalmol.2024.1269.
Heier JS, Lad EM, Holz FG, Rosenfeld PJ, Guymer RH, Boyer D, Grossi F, Baumal CR, Korobelnik JF, Slakter JS, Waheed NK, Metlapally R, Pearce I, Steinle N, Francone AA, Hu A, Lally DR, Deschatelets P, Francois C, Bliss C, Staurenghi G, Mones J, Singh RP, Ribeiro R, Wykoff CC; OAKS and DERBY study investigators. Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials. Lancet. 2023 Oct 21;402(10411):1434-1448. doi: 10.1016/S0140-6736(23)01520-9.
Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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APL2-303
Identifier Type: -
Identifier Source: org_study_id
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