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Geographic AtrophyPhase 2FILLYNCT02503332

Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy

A Phase 2 study testing Pegcetacoplan in people with Geographic Atrophy, run by Apellis Pharmaceuticals, Inc. at 46 sites in 3 countries.

Completed

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

Official record
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Trial summary

The primary objectives of the study are to assess the safety, tolerability and evidence of activity of multiple intravitreal (IVT) injections of pegcetacoplan in subjects with Geographic Atrophy associated with Age-Related Macular Degeneration (AMD). Read more about this study

Who is it for?
Ages 50 years and older
Conditions the study is for
Geographic Atrophy
What is being tested?
Pegcetacoplan
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
246 participants
Where
46 sitesin 3 countries
Study dates
24 Sep 2015to 17 Jan 2018
Phase
Phase 2
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 50 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Inclusion Criteria: Unless specified otherwise, ocular specific inclusion criteria apply to the study eye only.

  1. Male or Female.
  2. Age greater than or equal to 50 years.
  3. BCVA of 20/320 (Snellen equivalent) or better using ETDRS charts.
  4. Diagnosis of GA of the macula secondary to age-related macular degeneration, confirmed within 14 days prior to randomization by the central reading center (CRC) using Fundus Autofluorescence (FAF) images, as well as the following criteria:
  5. Total GA area must be ≥ 2.5 and ≤ 17.5 mm2 (1 and 7 disk areas [DA] respectively), determined by screening images of FAF.
  6. If GA is multifocal, at least one focal lesion must be ≥ 1.25 mm2 (0.5 DA).
  7. GA can be completely visualized on the macula centered image.
  8. GA must be able to be photographed in its entirety.
  9. GA must be able to be measured separately from any areas of peripapillary atrophy as assessed by the CRC.
  10. Presence of any pattern of hyperautofluorescence in the junctional zone of GA. Absence of hyperautoflouorescence (i.e. pattern = none) is exclusionary. See Holz et al. 2007.1
  11. Female subjects must be:
  12. Women of non-child-bearing potential (WONCBP), or
  13. Women of child-bearing potential (WOCBP) with a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study.
  14. 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.
  15. Willing and able to give informed consent.

Exclusion Criteria: Unless specified otherwise, ocular specific inclusion criteria apply to the study eye only.

  1. GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy.
  2. Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm.
  3. Any history or current evidence of exudative ("wet") AMD including any evidence of retinal pigment epithelium rips or evidence of neovascularization anywhere in the retina based on fluorescein angiogram as assessed by the CRC.
  4. Retinal disease other than AMD; however, benign conditions of the vitreous or peripheral retina are not exclusionary (i.e. pavingstone degeneration).
  5. Any ophthalmologic condition that reduces the clarity of the media and that, in the opinion of the Investigator interferes with ophthalmologic examination (e.g. advanced cataract or corneal abnormalities).
  6. Any ophthalmologic condition that prevents adequate imaging of the retina judged by the site or CRC.
  7. Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
  8. Aphakia or absence of the posterior capsule. Previous violation of the posterior capsule is also excluded unless it occurred as a result of yttrium aluminum garnet (YAG) laser posterior capsulotomy in association with prior posterior chamber intraocular lens implantation and at least 60 days prior to Day 0.
  9. Any ophthalmic condition that may require surgery during the study period.
  10. Any contraindication to IVT injection including current ocular or periocular infection.
  11. History of uveitis or endophthalmitis.
  12. History of IVT injection at any time.
  13. Participation in another interventional clinical study, or use of any experimental treatment for AMD or any other investigational new drug within 6 weeks or 5 half-lives of the active (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.
  14. Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the treatment period unlikely, or in general a poor medical risk because of other systemic diseases or active uncontrolled infections.
  15. 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.
  16. Hypersensitivity to fluorescein.

Trial locations and contacts

46 sites in 3 countries.

46 sites in 3 countries

United States35 sites

  • Retina Research Institute of Texas

    Abilene, Texas, 79606

  • South East Retina

    Augusta, Georgia, 30909

  • Retina Research Center

    Austin, Texas, 78705

  • Elman Research

    Baltimore, Maryland, 21237

  • Retina Vitreous Asociates Mdical Goup

    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

    Charlotte, North Carolina, 28210

  • Cleveland Clinic Foundation/ Cole Eye Institute

    Cleveland, Ohio, 44106

  • Retina Associates of Cleveland

    Cleveland, Ohio, 44122

  • Duke University, Duke Eye Center

    Durham, North Carolina, 27705

  • Retina Health Center

    Fort Myers, Florida, 33907

  • Associated Retinal Consultants PC

    Grand Rapids, Michigan, 45946

  • Illinois Retina Associates

    Harvey, Illinois, 60426

  • Retina Consultants of Houston

    Houston, Texas, 77030

  • Midwest Eye Institute

    Indianapolis, Indiana, 46290

  • The Gavin Herbert Eye Institute/UC Irvine

    Irvine, California, 92697

  • University of Southern California - USC Eye Institute

    Los Angeles, California, 90033

  • Valley Retina Institute, PA

    McAllen, Texas, 75803

  • Bascom Palmer Eye Institute

    Miami, Florida, 33136

  • Retina Speciality Institute

    Mobile, Alabama, 36606

  • Tennessee Retina, PC

    Nashville, Tennessee, 37203

  • New England Retina Associates

    New London, Connecticut, 06320

  • Vitreous Retina Macula Consultants of New York

    New York, New York, 10022

  • Byers Eye Institute at Stanford, Stanford School of Medicine

    Palo Alto, California, 94303

  • Mid Atlantic

    Philadelphia, Pennsylvania, 19006

  • Retinal Research Institute

    Phoenix, Arizona, 85014

  • Retina Specialists

    Plano, Texas, 75093

  • Eyesight Opthalmic Services PA

    Portsmouth, New Hampshire, 03801

  • Black Hills Regional Eye Institute

    Rapid City, South Dakota, 57701

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of Utah

    Salt Lake City, Utah, 84132

  • Charlotte Eye Ear Nose and Throat Associates

    Statesville, North Carolina, 28677

  • Retina Consultants of Houston (The Woodlands)

    The Woodlands, Texas, 77384

  • Associated Retinal Consultants, PC

    Traverse City, Michigan, 49586

Other trials in United States

Australia9 sites

  • Royal Victorian Eye and Ear Hospital

    East Melbourne, Victoria, 3002

  • Hobart eye Surgeons

    Hobart, Tasmania, 7002

  • Center for Eye Research Australia

    Melbourne, Victoria, 3002

  • Lions Eye Institute

    Nedlands, Western Australia, 6009

  • Marsden Eye Specialists

    Parramatta, New South Wales, 2150

  • Tasmanian Eye Institute

    South Launceston, Tasmania, 7249

  • Save Sight Institute, Sydney Eye Hospital

    Sydney, New South Wales, 2000

  • Sydney Retina Clinic and Day Surgery

    Sydney, New South Wales, 2000

  • Sydney West Retina

    Westmead, New South Wales, 2145

Other trials in Australia

New Zealand2 sites

  • Southern Eye Specialists

    Merivale, Christchurch, 8014

  • Auckland Eye

    Remuera, Auckland, 1050

Other trials in New Zealand

About this study

Official title: A Phase II, Multicenter, Randomized, Single-Masked, Sham-Controlled Study of Safety, Tolerability and Evidence of Activity of Intravitreal APL-2 Therapy in Patients With Geographic Atrophy (GA)

The primary objectives of the study are to assess the safety, tolerability and evidence of activity of multiple intravitreal (IVT) injections of pegcetacoplan in subjects with Geographic Atrophy associated with Age-Related Macular Degeneration (AMD).

Read the detailed description

This is a Phase II, prospective, multicenter, randomized, single-masked, sham-controlled study to assess the safety, tolerability and evidence of activity of multiple IVT injections of pegcetacoplan in subjects with GA secondary to Age-Related Macular Degeneration.

The study will randomize approximately 240 subjects to obtain at least 200 evaluable subjects across 40 multinational sites.

Subjects will be randomized in a 2:2:1:1 manner to receive pegcetacoplan Monthly (AM), pegcetacoplan Every-Other-Month (AEOM), Sham injection Monthly (SM) or Sham injection Every-Other-Month (SEOM), respectively.

All subjects will return to the clinical site on Day 7 to assess acute safety after the first injection. After that, subjects in the monthly groups will return to the clinical site for additional pegcetacoplan (or Sham) injections and study procedures every month until Month 12. Subjects in the Every-Other-Month groups will return to the clinical site for additional pegcetacoplan (or Sham) injections and study procedures every two months until Month 12. All subjects will return for follow-up visits on Months 15 and 18 (3 and 6 months after last injection, respectively).

Being tested
Pegcetacoplan, 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.

  • PegcetacoplanDrug

    Also called APL-2

  • Sham ProcedureOther

Study groups in NCT02503332
Study groupRoleWhat they receive
Pegcetacoplan 15 mg/100 µL Monthly for 12 monthsExperimentalA single dose of 15 mg pegcetacoplan/100 µL will be administered via intravitreal injection in this study. Subjects will receive an injection every month for 12 consecutive months.
Receives: Drug: Pegcetacoplan
Pegcetacoplan 15 mg/100 µL EOM for 12 monthsExperimentalA single dose of 15 mg pegcetacoplan/100 µL will be administered via intravitreal injection in this study. Subjects will receive an injection every other month (EOM) for 12 consecutive months.
Receives: Drug: Pegcetacoplan
Sham Monthly for 12 monthsSham comparatorSubjects will receive a Sham procedure every month for 12 consecutive months.
Receives: Other: Sham Procedure
Sham EOM for 12 monthsSham comparatorSubjects will receive a Sham procedure every other month (EOM) for 12 consecutive months.
Receives: Other: Sham Procedure

What is being measured

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

  1. Least Square (LS) Mean Change From Baseline in Square Root GA Lesion Size in the Study Eye at Month 12

    The square root GA lesion size (i.e. transformed area of GA) was measured by FAF photographs. Baseline was defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (screening) and Month 12.

  2. Number of Subjects With Treatment Emergent Adverse Events (TEAEs) in the Study Eye, Including by Severity

    A TEAE was defined as any adverse event (AE) that commenced or worsened on or after time of first study drug administration up to 60 days beyond last dose of study drug. A treatment-related TEAE was defined as a TEAE with a relationship to study drug of possibly related or probably related or not reported. Severity of TEAEs were categorized as mild; moderate; severe; life-threatening or death related to TEAE, according to Common Terminology Criteria for AEs v4.03. A TEAE of special interest (TEAESI) was defined as a TEAE of scientific and medical concern specific to pegcetacoplan, whether serious or non-serious.

    Measured: From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).

Secondary outcomes (6)

  1. LS Mean Change From Baseline in Untransformed GA Lesion Size in the Study Eye at Month 12

    The untransformed area of GA was measured by FAF. Baseline is defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (Day 1) and Month 12.

  2. LS Mean Change From Baseline in Best-Corrected Visual Acuity (BCVA) Score of the Study Eye at Month 12

    The BCVA letter score was determined using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart assessed at a starting distance of 4 meters. The score ranges from 0 to 100 letters, lower number indicating reduced visual acuity; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.

    Measured: Baseline (Day 1) and Month 12.

  3. LS Mean Change From Baseline in Low Luminance BCVA (LL-BCVA) Score in the Study Eye at Month 12

    The LL-BCVA was measured by placing a 2.0-log-unit neutral density filter over the best correction and having the participant read the normally illuminated ETDRS chart. The score ranges from 0 to 100 letters, lower number indicating worse vision; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.

    Measured: Baseline (Day 1) and Month 12.

  4. LS Mean Change From Baseline in Low Luminance VA (LL-VA) Deficit Score in the Study Eye at Month 12

    The LL-VA deficit score is calculated as BCVA score minus LL-BCVA score. The LL-VA deficit score ranges from 0 to 100 letters, lower number indicating worse deficit.

    Measured: Baseline (Day 1) and Month 12.

  5. LS Mean Change From Baseline in Distance of GA Lesion From the Fovea (Foveal Encroachment) in the Study Eye at Month 12

    The foveal encroachment in the study eye was measured by FAF. Baseline is defined as the last available, non-missing observation prior to first study drug administration.

    Measured: Baseline (Day 1) and Month 12.

  6. Number of Subjects With Any Macular Neovascularization (MNV) TEAEs in the Study Eye

    The number of subjects with any MNV TEAEs in the study eye was identified via clinical review of all ocular TEAEs.

    Measured: From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).

Timeline

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

  1. First posted20 Jul 2015 (estimated)
  2. Study start24 Sep 2015
  3. Primary completion14 Jul 2017
  4. Study completion17 Jan 2018

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Single group
Masking
Single blind
Who is masked
Participant
Enrolment
246 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02503332
Sponsor's protocol ID
POT-CP121614
Acronym
FILLY
Status last verified
Sep 2020
First submitted
14 Jul 2015
Last updated
6 Oct 2020
Data monitoring committee
Yes

Publications

  • Reiter GS, Lachinov D, Buhl W, Weigert G, Grechenig C, Mai J, Bogunovic H, Schmidt-Erfurth U. A Novel Management Challenge in Age-Related Macular Degeneration: Artificial Intelligence and Expert Prediction of Geographic Atrophy. Ophthalmol Retina. 2025 May;9(5):421-430. doi: 10.1016/j.oret.2024.10.029. Epub 2024 Nov 9.

    PubMed 39522752

  • Hatcher KA, Shah BK, Burch M, Kondapalli SSA, Baumal CR. Outer Retinal Tubulation in Geographic Atrophy. Ophthalmic Surg Lasers Imaging Retina. 2024 Aug;55(8):448-451. doi: 10.3928/23258160-20240411-02. Epub 2024 May 1.

    PubMed 38752918

  • Fu DJ, Lipkova V, Liefers B, Glinton S, Faes L, McKeown A, Scheibler L, Pontikos N, Patel PJ, Zhang G, Keane PA, Balaskas K. Evaluating the Effects of C3 Inhibition on Geographic Atrophy Progression from Deep-Learning OCT Quantification: A Split-Person Study. Ophthalmol Ther. 2023 Dec;12(6):3143-3158. doi: 10.1007/s40123-023-00798-7. Epub 2023 Sep 16.

    PubMed 37715860

  • 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

  • Pfau M, Schmitz-Valckenberg S, Ribeiro R, Safaei R, McKeown A, Fleckenstein M, Holz FG. Association of complement C3 inhibitor pegcetacoplan with reduced photoreceptor degeneration beyond areas of geographic atrophy. Sci Rep. 2022 Oct 25;12(1):17870. doi: 10.1038/s41598-022-22404-9.

    PubMed 36284220

  • Vogl WD, Riedl S, Mai J, Reiter GS, Lachinov D, Bogunovic H, Schmidt-Erfurth U. Predicting Topographic Disease Progression and Treatment Response of Pegcetacoplan in Geographic Atrophy Quantified by Deep Learning. Ophthalmol Retina. 2023 Jan;7(1):4-13. doi: 10.1016/j.oret.2022.08.003. Epub 2022 Aug 7.

    PubMed 35948209

  • Liao DS, Metlapally R, Joshi P. Pegcetacoplan treatment for geographic atrophy due to age-related macular degeneration: a plain language summary of the FILLY study. Immunotherapy. 2022 Sep;14(13):995-1006. doi: 10.2217/imt-2022-0078. Epub 2022 Jul 21.

    PubMed 35860926

  • Nittala MG, Metlapally R, Ip M, Chakravarthy U, Holz FG, Staurenghi G, Waheed N, Velaga SB, Lindenberg S, Karamat A, Koester J, Ribeiro R, Sadda S. Association of Pegcetacoplan With Progression of Incomplete Retinal Pigment Epithelium and Outer Retinal Atrophy in Age-Related Macular Degeneration: A Post Hoc Analysis of the FILLY Randomized Clinical Trial. JAMA Ophthalmol. 2022 Mar 1;140(3):243-249. doi: 10.1001/jamaophthalmol.2021.6067.

    PubMed 35113137

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pegcetacoplan in people with Geographic Atrophy, run by Apellis Pharmaceuticals, Inc. at 46 sites in 3 countries.

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

Who may be eligible to take part?

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 6 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 46 sites in United States, Australia, New Zealand. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 6 Oct 2020). 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 Square (LS) Mean Change From Baseline in Square Root GA Lesion Size in the Study Eye at Month 12 (time frame: Baseline (screening) and Month 12.). The study lists 2 primary outcomes in all. 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 NCT02503332, which also lists 8 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 6 Oct 2020.

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