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Geographic AtrophyPhase 3NCT03525600

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

A Phase 3 study testing APL-2 in people with Geographic Atrophy, run by Apellis Pharmaceuticals, Inc. at 141 sites in 15 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a 24-month, Phase III, multicenter, randomized, double-masked, sham-injection controlled study to assess the efficacy and safety of multiple IVT injections of APL-2 in subjects with GA secondary to AMD. Read more about this study

Who is it for?
Ages 60 years and older
Conditions the study is for
Geographic Atrophy
What is being tested?
APL-2
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
621 participants
Where
141 sitesin 15 countries
Study dates
31 Aug 2018to 20 Jun 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 60 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • The study eye must meet all inclusion criteria. If both eyes meet the inclusion criteria, the eye with the worst visual acuity at the screening visit will be designated as the study eye. If both eyes have the same visual acuity, the right eye will be selected as the study eye.
  • Ocular- specific inclusion criteria apply to the study eye only, unless otherwise specified.
  • Age ≥ 60 years.
  • 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:
Show all 16Show fewer
  • 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.

Who cannot join

  • Ocular specific exclusion criteria apply to the study eye only, unless otherwise specified.
  • GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye.
  • 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.
Show all 17Show fewer
  • 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.

Trial locations and contacts

141 sites in 15 countries.

141 sites in 15 countries

United States73 sites

  • Retina Research Institute of Texas

    Abilene, Texas, 79606

  • Southwest Retina Specialists

    Amarillo, Texas, 79106

  • Retina Institute of California dba Acuity Eye Grp

    Arcadia, California, 91007

  • Western Carolina Retinal Associates

    Asheville, North Carolina, 28803

  • Southeast Retina Center, PC

    Augusta, Georgia, 30909

  • Retina Consultants of Austin (Retina Research Center)

    Austin, Texas, 78705

  • California Retina Consultants

    Bakersfield, California, 93309

  • Elman Retina Group, PA

    Baltimore, Maryland, 21236

  • Vitreoretinal Associates of Washington

    Bellevue, Washington, 98004

  • Retina Vitreous Associates Medical Group

    Beverly Hills, California, 90211

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114

  • Florida Eye Microsurgical Institute, Inc.

    Boynton Beach, Florida, 33426

  • Charlotte Eye Ear Nose and Throat Associates, PS

    Charlotte, North Carolina, 28210

  • University of Virginia

    Charlottesville, Virginia, 22903

  • Retina Associates of Cleveland, Inc.

    Cleveland, Ohio, 44122

  • Retina Associates of Cleveland, Inc

    Cleveland, Ohio, 44122

  • Retina Associates of Cleveland

    Cleveland, Ohio, 44130

  • Cleveland Clinic, Cole Eye Institute

    Cleveland, Ohio, 44195

  • The Ohio State University

    Columbus, Ohio, 43212

  • Danbury Eye Physicians & Surgeons, P.C. - Danbury

    Danbury, Connecticut, 06810

  • The Retina Partners

    Encino, California, 91436

  • The Retina Group of Washington

    Fairfax, Virginia, 22031

  • Pinnacle Research Institute

    Fort Lauderdale, Florida, 33309

  • Retina Health Center

    Fort Myers, Florida, 33907

  • Associated Retinal Consultants, P.C

    Grand Rapids, Michigan, 49546

  • Retina Specialists of Michigan / Foundation for Vision

    Grand Rapids, Michigan, 49546

  • Long Island Vitreoretinal Consultants

    Great Neck, New York, 11021

  • Cumberland Valley Retina Consultants, PC

    Hagerstown, Maryland, 21740

  • Mid Atlantic Retina Specialists

    Hagerstown, Maryland, 21740

  • New England Retina Associates

    Hamden, Connecticut, 06518

  • Graystone Eye

    Hickory, North Carolina, 28602

  • Retina Consultants of Houston, PA

    Houston, Texas, 77030

  • Midwest Eye Institute

    Indianapolis, Indiana, 46290

  • The Gavin Herbert Eye Institute/UC Irvine

    Irvine, California, 92697

  • Sabates Eye Center

    Leawood, Kansas, 66211

  • University of Wisconsin

    Madison, Wisconsin, 53715

  • Georgia Retina

    Marietta, Georgia, 30060

  • Valley Retina Institute, PA

    McAllen, Texas, 78503

  • Northern California Retina Vitreous Associates

    Mountain View, California, 94040

  • Retina Associates of Utah, PC

    Murray, Utah, 84107

  • Tennessee Retina, PC

    Nashville, Tennessee, 37203

  • Vitreous Retina Macula Consultants of NY

    New York, New York, 10022

  • Bascom Palmer Eye Institute

    Palm Beach Gardens, Florida, 33418

  • Retina Institute of California Medical Group

    Palm Desert, California, 92260

  • Byers Eye Institute at Standford, Stanford School of Medicine

    Palo Alto, California, 94303

  • Mid Atlantic Retina

    Philadelphia, Pennsylvania, 19006

  • Retinal Research Institute

    Phoenix, Arizona, 85014

  • Associated Retina Consultants, Ltd

    Phoenix, Arizona, 85020

  • Eye Associates of Pinellas

    Pinellas Park, Florida, 33782

  • Retina Northwest, PC

    Portland, Oregon, 97221

  • Eye Health Northwest

    Portland, Oregon, 97225

  • Retina Consultants San Diego

    Poway, California, 92064

  • Black Hills Regional Eye Institute

    Rapid City, South Dakota, 57701

  • Retina Consultants of Southern California

    Redlands, California, 92374

  • Sierra Eye Associates

    Reno, Nevada, 89503

  • Medical Center Ophthalmology Associates

    San Antonio, Texas, 78240

  • Retinal Consultants of San Antonio

    San Antonio, Texas, 78240

  • California Retina Consultants

    Santa Barbara, California, 93103

  • Retina Consultants of Michigan

    Southfield, Michigan, 48034

  • Spokane Eye Clinical Research

    Spokane, Washington, 99204

  • New England Retina Consultants, PC

    Springfield, Massachusetts, 01107

  • East Florida Eye Institute

    Stuart, Florida, 34994

  • Southern Vitreoretinal Associates

    Tallahassee, Florida, 32308

  • Retina Associates of Florida

    Tampa, Florida, 33609

  • University of South Florida (USF) Eye Institute

    Tampa, Florida, 33612

  • Retina Associates of New Jersey (NJ Retina)

    Teaneck, New Jersey, 07666

  • Retina Consultants of Houston

    The Woodlands, Texas, 77384

  • Retina Specialists

    Towson, Maryland, 21204

  • Associated Retinal Consultants PC

    Traverse City, Michigan, 49686

  • Bay Area Retina Associates

    Walnut Creek, California, 94598

  • Virginia Retina Center

    Warrenton, Virginia, 20186

  • Retina Group of New England,PC

    Waterford, Connecticut, 06385

  • Retina Associates of Cleveland, Inc.

    Youngstown, Ohio, 44505

Other trials in United States

Argentina10 sites

  • Fundacion Zambrano

    Buenos Aires, Buenos Aires F.D., C1023 AAQ

  • Organizacion Medica de investigacion

    Buenos Aires, C1015 ABO

  • Centro Oftalmologico Dr Charles

    Buenos Aires, C1116ABA

  • Diagnostico Ocular

    Buenos Aires, C1425BGE

  • Centro Privado de Ojos Romagosa SA

    Córdoba, X5000AAJ

  • Instituto Oftalmologico de Cordoba

    Córdoba, X5000III

  • Microcirugia Ocular

    El Rosario, Corrientes Province, 2000

  • Oftar Mendoza SRL

    Mendoza, 5500

  • Grupo Laser Vision

    Rosario, Santa Fe Province, S2000 AZH

  • Oftalmologos Especialistas

    Rosario, 1288

Other trials in Argentina

United Kingdom10 sites

  • Bristol Eye Hospital

    Bristol, BS1 2LX

  • Eye Clinic, Acre Mill Outpatients, Huddersfield Royal Infirmary

    Huddersfield, West Yorkshire, HD3 3EA

  • St James's University Hospital

    Leeds, LS9 7TF

  • Leicester Royal Infirmary

    Leicester, LEI 5WW

  • Moorfields Eye Hospital NHS Foundation Trust

    London, England, EC1V 2PD

  • London North West University Hospital Trust

    London, NW10 7NS

  • King's College Hospital NHS Trust

    London, SE5 9RS

  • Oxford Eye Hospital

    Oxford, OX3 9DU

  • Salisbury NHS Foundation Trust

    Salisbury, SP2 8BJ

  • Sunderland Eye Infirmary

    Sunderland, SR2 9HP

Other trials in United Kingdom

France7 sites

  • Centre Hospitalier Intercommunal de Créteil

    Créteil

  • Hopital de la Croix-Rousse

    Lyon, 69004

  • Centre Monticelli Paradis

    Marseille, 13008

  • CHU de Nantes - Hotel Dieu

    Nantes, 44093

  • Centre Ophtalmologique de l´Odéon

    Paris, 75006

  • Centre Ophthalmologique Saint-Exupery

    Saint-Cyr-sur-Loire, 37540

  • Maison Rouge Ophthalmologic Center

    Strasbourg, 67000

Other trials in France

Brazil6 sites

  • Instituto da Visão - Hospital de Olhos Ltda

    Belo Horizonte, 32150-274

  • Clinica Oftalmologica Sao Lucas

    Osasco, São Paulo, 06010-130

  • Hospital De Clinicas De Porto Alegre

    Porto Alegre, 90036-903

  • IPEPO - Instituto Da Visao

    São Paulo, Vila Clementino, 04038-032

  • UNIFESP - Federal University

    São Paulo, 04021-001

  • Clinica Ocular Oftalmologia LTDA

    Vitória, Espírito Santo, 29055-45

Other trials in Brazil

Germany6 sites

  • Universitäts-Augenklinik Bonn

    Bonn, 53127

  • University Hospital Cologne

    Cologne, 50937

  • Klinikum der Stadt Ludwigshafen gGmbH

    Ludwigshafen, 67063

  • Klinikum rechts der Isar

    München, 81675

  • Universitätsklinikum Regensburg

    Regensburg, 93053

  • University Hospital Würzburg

    Würzburg, 97080

Other trials in Germany

Israel6 sites

  • Shamir Medical Center

    Be’er Ya‘aqov, 7030000

  • Rambam Medical Center

    Haifa, 3109601

  • Meir Medical Center

    Kfar Saba, 4428164

  • Rabin Medical Center

    Petah Tikva, 49100

  • Kaplan Medical Center

    Rehovot, 76100

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 64239

Other trials in Israel

Canada4 sites

  • Ivey Eye Institute

    London, Ontario, N6A

  • University of Ottawa Eye Institute

    Ottawa, Ontario, K1H 8L6

  • Retina Centre of Ottawa

    Ottawa, K2B 7E9

  • DRY AMD Clinic - St. Michael's Hospital

    Toronto, Ontario, M5C 1R6

Other trials in Canada

Czechia4 sites

  • University Hospital Kralovske Vinochrady

    Brno-Vinohrady, 100 34

  • Fakultní nemocnice Ostrava

    Ostrava-Poruba, 708 52

  • OFTEX Eye Clinic

    Pardubice, 53002

  • AXON Clinical, S.R.O.

    Prague, 150 00

Other trials in Czechia

Poland4 sites

  • Oftalmika Eye Hospital

    Bydgoszcz, 85-631

  • Jasne Blonia Eye Clinic

    Lodz, 91-134

  • Centrum Diagnostyki i Mikrochirurgii Oka - LENS

    Olsztyn, 10-424

  • Centrum Medyczne UNO-MED

    Tarnów, 33-100

Other trials in Poland

Australia3 sites

  • Centre for Eye Research Australia

    East Melbourne, Victoria, 3002

  • Retina and Eye Consultants

    Hurstville, NSW 2220

  • Sydney Retina

    Sydney, New South Wales, 2000

Other trials in Australia

Spain3 sites

  • Centro Médico Teknon

    Barcelona, 08022

  • Instituto Oftalmologico Gómez-Ulla

    Santiago de Compostela, 15706

  • Hospital Universitario Rio Hortega

    Valladolid, 47006

Other trials in Spain

Italy2 sites

  • Ospedale San Raffaele

    Milan, 20132

  • Luigi Sacco Hospital

    Milan, 20157

Other trials in Italy

New Zealand2 sites

  • Retina Specialist

    Auckland, 1052

  • Hamilton Eye Clinic

    Hamilton, 3204

Other trials in New Zealand

Puerto Rico1 site

  • Emanuelli Research and Development Center

    Arecibo, 00612

Other trials in Puerto Rico

About this study

Official title: A Phase 3, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal Pegcetacoplan Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

This is a 24-month, Phase III, multicenter, randomized, double-masked, sham-injection controlled study to assess the efficacy and safety of multiple IVT injections of APL-2 in subjects with GA secondary to AMD.

Being tested
APL-2, Sham Procedure
Medical subject headings
Geographic Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • APL-2Drug

    Complement (C3) Inhibitor

  • APL-2Drug

    Complement (C3) Inhibitor

  • Sham ProcedureOther

    Subjects will receive a Sham procedure every month

  • Sham ProcedureOther

    Subjects will receive a Sham procedure every other month

Study groups in NCT03525600
Study groupRoleWhat they receive
APL-2 15mg 0.1 mL monthly for 24 monthsExperimentalA 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
Receives: Drug: APL-2
APL-2 15mg 0.1 mL EOM for 24 monthsExperimentalA 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
Receives: Drug: APL-2
Sham Procedure Monthly for 24 monthsExperimentalSham Procedure for 24 months
Receives: Other: Sham Procedure
Sham Procedure Every Other Month for 24 monthsExperimentalSham Procedure every other month for 24 months
Receives: Other: Sham Procedure

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Least Squares (LS) Mean Change From Baseline in Total Area of GA Lesions in the Study Eye at Month 12

    The GA lesion area was measured by a quantified central reading center based on FAF images. LS mean was calculated using a mixed effect model for repeated measure (MMRM) model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (screening) and Month 12

Secondary outcomes (5)

  1. LS Mean Change From Baseline in the Total Area of GA Lesions in the Study Eye at Month 24

    The GA lesion area was measured by a quantified central reading center based on FAF images. LS mean was calculated using a MMRM model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (screening) and Month 24

  2. Mean Change in Total Area of GA Lesions in the Study Eye Through Month 24

    The mean change in GA lesion area through Month 24 was measured by assuming a piecewise linear trend in time with knots by FAF images at Months 6, 12, and 18 and was calculated using a MMRM model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: From Baseline (screening) through Month 24

  3. LS Mean Change From Baseline in Monocular Maximum Reading Speed of the Study Eye at Month 24

    The maximum reading speed of the study eye was calculated per Minnesota Low-Vision Reading Test (MNREAD) or Radner Reading Charts user manuals, with no adjustment for reading inaccuracy. An additional step to cap resulting reading speed values at a maximum of 300 words per minute (wpm) was implemented. Maximum reading speed was calculated as the mean of the 3 highest non-zero reading speeds (or 2, or 1 value, as available), except when all wpm were calculated as 0 then the maximum reading speed was calculated as 0. LS mean was calculated using a MMRM model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (screening) and Month 24

  4. LS Mean Change From Baseline in Mean Functional Reading Independence (FRI) Index Score at Month 24

    The FRI was an interviewer-administered questionnaire with 7 items on functional reading activities most relevant to GA AMD subjects. It had 1 total index score. For each FRI Index reading activity performed in the past 7 days, subjects were asked about the extent to which they required assistance beyond eyeglasses/contact lenses, including the use of low-vision aids, adjustments in the activity, or help from another subject. Mean FRI Index scores ranged from 1 (unable to do independently) to 4 (totally independent), with higher scores indicating higher functional reading independence. A negative change from baseline indicated a decrease in the FRI; disease worsening. LS mean was calculated using a MMRM model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (screening) and Month 24

  5. LS Mean Change From Baseline in Normal-Luminance Best-Corrected Visual Acuity (NL-BCVA) Score of the Study Eye at Month 24

    The NL-BCVA was assessed by early treatment diabetic retinopathy study (ETDRS) chart prior to dilating the eyes at a starting distance of 4 meters and ranged from 0 (least score) to 100 (best score). If the 4-meter score was >19 letters read correctly, the visual acuity score was the sum of total letters correctly read at 4 meters plus the addition of 30. If the 4-meter score was ≤19 letters read correctly, the visual acuity score was the sum of total letters read correctly at 4 meters and total letters read correctly at the 1-meter distance. If no letters were read correctly at either the 4-meter distance or the 1-meter distance, the visual acuity score was 0. A positive change in the value indicated improvement in visual acuity. LS mean was calculated using a MMRM model. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (screening) and Month 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 May 2018
  2. Study start31 Aug 2018
  3. Primary completion21 Jun 2021
  4. Study completion20 Jun 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
621 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03525600
Sponsor's protocol ID
APL2-303
Status last verified
May 2023
First submitted
20 Apr 2018
Last updated
18 Jun 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 40388106

  • 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.

    PubMed 40280279

  • Wykoff CC, Lad EM, Heier JS. Syfovre Approval for Geographic Atrophy. JAMA Ophthalmol. 2024 Jul 1;142(7):685-686. doi: 10.1001/jamaophthalmol.2024.1754. No abstract available.

    PubMed 38814626

  • 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.

    PubMed 38722644

  • 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.

    PubMed 37865470

  • 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.

    PubMed 37314061

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing APL-2 in people with Geographic Atrophy, run by Apellis Pharmaceuticals, Inc. at 141 sites in 15 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 141 sites in United States, Argentina, United Kingdom, France, Brazil and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Least Squares (LS) Mean Change From Baseline in Total Area of GA Lesions in the Study Eye at Month 12 (time frame: Baseline (screening) and Month 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03525600, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2023.

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