A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration

Study Overview

• Status: Active, not recruiting
• Conditions: Geographic Atrophy
• Intervention / Treatment: Drug: Pegcetacoplan

• Study Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

Study Locations

• United States
  • California
    • Bakersfield, California, United States, 93309
      • California Retina Consultants (01-026)
    • Beverly Hills, California, United States, 90211
      • Retina-Vitreous Associates Medical Group (01-020)
    • Fullerton, California, United States, 92835
      • Illinois Retina Associates (01-035)
    • Modesto, California, United States, 95356
      • Retinal Consultants Medical Group Inc (01-008)
    • Poway, California, United States, 92064
      • Retina Consultants San Diego Inc. (01-018)
    • Santa Barbara, California, United States, 93103
      • California Retina Consultants (01-027)
    • Walnut Creek, California, United States, 94598
      • Bay Area Retina Associates (01-005)
  • Colorado
    • Lakewood, Colorado, United States, 80228
      • Colorado Retina Associates, PLLC (01-011)
  • Florida
    • Pensacola, Florida, United States, 32503
      • Retina Specialty Institute (01-019)
  • Georgia
    • Augusta, Georgia, United States, 30909
      • South East Retina Center, PC (01-030)
    • Marietta, Georgia, United States, 30060
      • Georgia Retina (01-032)
  • Illinois
    • Oak Park, Illinois, United States, 60304
      • Illinois Retina Associates (01-037)
  • Maryland
    • Baltimore, Maryland, United States, 21209
      • The Retina Care Center (01-023)
    • Hagerstown, Maryland, United States, 21740
      • Cumberland Valley Retina Consultants, P.C. (01-002)
    • Hagerstown, Maryland, United States, 21740
      • Mid Atlantic Retina Specialist (01-029)
  • Minnesota
    • Edina, Minnesota, United States, 55435
      • Retina Consultants of Minnesota, PLLC (01-025)
  • New Jersey
    • Cherry Hill, New Jersey, United States, 08034
      • Mid Atlantic Retina Research (01-014)
    • Cherry Hill, New Jersey, United States, 08906
      • Mid Atlantic Retina (01-031)
  • New York
    • Oceanside, New York, United States, 11572
      • OCLI Research Department (01-007)
    • Westbury, New York, United States, 11590
      • Long Island Vitreoretinal Consultants (01-013)
  • North Carolina
    • Asheville, North Carolina, United States, 28803
      • Western Carolina Retinal Associates, PA (01-009)
  • Ohio
    • Cleveland, Ohio, United States, 44122
      • Retina Associates of Cleveland, Inc. (01-017)
    • Cleveland, Ohio, United States, 44130
      • Retina Associates of Cleveland, Inc. (01-022)
    • Youngstown, Ohio, United States, 44505
      • Retina Associates of Cleveland, Inc. (01-016)
  • Pennsylvania
    • Bethlehem, Pennsylvania, United States, 18017
      • Mid Atlantic Retina (01-001)
    • York, Pennsylvania, United States, 17402
      • The Retina Care Center (01-034) - Satellite Site
  • South Dakota
    • Rapid City, South Dakota, United States, 57701
      • Black Hills Regional Eye Institute (01-010)
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Tennessee Retina (01-033)
  • Texas
    • Abilene, Texas, United States, 79606
      • Integrated Clinical Research (01-024)
    • Bellaire, Texas, United States, 77401
      • Retina Consultants of Texas (01-004)
    • Dallas, Texas, United States, 75231
      • Texas Retina Associates (01-028)
    • The Woodlands, Texas, United States, 77384
      • Retina Consultants of Texas (01-003)
  • Virginia
    • Fairfax, Virginia, United States, 22031
      • The Retina Group of Washington (01-021)
  • Washington
    • Bellevue, Washington, United States, 98004
      • Pacific Northwest Retina (01-036)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients residing in the United States who are 60 years or older who have geographic atrophy secondary to age-related macular degeneration

Description

Inclusion Criteria: Eyes are eligible to be included in the study only if all of the following criteria apply. Ocular-specific inclusion criteria apply to the treated eye(s), unless otherwise specified.

• Eyes that are naive to treatment with pegcetacoplan that are prescribed pegcetacoplan per routine clinical practice according to approved pegcetacoplan prescribing information (ie, the clinical decision to treat with pegcetacoplan must be made before study participation is discussed with the patient)
• Patient age ≥60 years
• Visual acuity better than 20/200 on Snellen chart
• Clinical diagnosis of GA of the macula secondary to AMD in one or both eyes as determined by the investigator using OCT and/or FAF imaging

• GA lesion meeting the following criteria as determined by the investigator's assessment of Spectralis OCT and/or FAF images at screening:

  1. Nonsubfoveal lesion(s)
  2. GA lesion visualized in its entirety on the macula-centered OCT image and not contiguous with any areas of peripapillary atrophy
  3. Presence of any pattern of hyperautofluorescence in the junctional zone of GA; absence of hyperautofluorescence (ie, pattern = none) exclusionary
• Adequate clarity of ocular media and adequate pupillary dilation and fixation to permit the collection of good quality images as determined by the investigator
• Patient willing and able to give informed consent and to comply with the study procedures and assessments

Exclusion Criteria: Ocular-specific exclusion criteria apply to the study eye(s) 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
• Active, suspected, or history of intraocular inflammation in either eye at screening or on day 1
• Any history of or active choroidal neovascularization associated with AMD or any other cause, including any evidence of retinal pigment epithelial tears or rips in SD OCT imaging
• Presence of an active ocular disease that in the opinion of the investigator compromises or confounds visual function, including but not limited to uveitis and other macular diseases (eg, clinically significant epiretinal membrane, full thickness macular hole, uncontrolled glaucoma/ocular hypertension). Conditions that in the opinion of the investigator are benign, such as peripheral retina dystrophy, are not exclusionary
• Any prior treatment with anti-VEGF agents
• Intraocular surgery (including lens replacement surgery) within 3 months prior to screening
• History of laser therapy in the macular region
• Aphakia or absence of the posterior capsule. Note: Yttrium aluminum garnet 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
• Current ocular or periocular infection in either eye
• Intravitreal medical device placement
• Participation in any prior or current systemic experimental treatment within 6 weeks or 5 half lives of the active ingredient (whichever is longer) prior to the start of study treatment or in any other investigational treatments specific to GA. 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 unlikely or would make the patient an unsafe study candidate
• Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to pegcetacoplan or any of the excipients in pegcetacoplan solution
• History or current use of brolucizumab and/or pharmacological treatments that gain approval for the treatment of GA

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Observation	Drug: Pegcetacoplan; Administered intravitreally (IVT) in eyes with geographic atrophy (GA); Other Names: Syfovre

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of ocular AEs in treated eyes overall and according to physician-directed treatment interval based on US prescribing information	Up to 36 months
Incidence of other events of interest	Up to 36 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Assess real-world treatment patterns and use of pegcetacoplan considering the proportion of participants with bilateral GA treated with pegcetacoplan in both eyes	Up to 36 months
If only one eye is treated, the criteria for determination of the eye to be treated	Up to 36 months
The treatment interval (mean days between injections) over time	Up to 36 months
The frequency of imaging on treated eyes (color fundus photography, spectral domain optical coherence tomography [SD-OCT], fundus autofluorescence, optical coherence tomography angiography [OCT-A	Up to 36 months
The treatment adherence to planned dosing frequency: the number of injections administered divided by the number of injections planned	Up to 36 months
The duration of persistence on treatment: continuous medication usage until a gap of ≥3 months	Up to 36 months
The treatment discontinuation and/or switching to other agents approved for treatment of GA	Up to 36 months
The management of new-onset exudative age-related macular degeneration (eAMD) events in eyes treated with pegcetacoplan in clinical practice	Up to 36 months
The proportion of treated eyes that develop eAMD that receive anti-vascular endothelial growth factor (VEGF) injections	Up to 36 months
The number of anti-VEGF injections per month from diagnosis of eAMD	Up to 36 months
The proportion of anti-VEGF injections given on same day in the same eye as pegcetacoplan	Up to 36 months

Collaborators and Investigators

• Sponsor: Apellis Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 28, 2023
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: October 18, 2023
• First Submitted That Met QC Criteria: November 29, 2023
• First Posted (Actual): December 8, 2023

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 28, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Geographic Atrophy; pegcetacoplan
• Other Study ID Numbers: APL2-GA-411

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No