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

Apellis Pharmaceuticals, Inc.34 sites in 1 country300 target enrollmentSeptember 28, 2023

ConditionsGeographic Atrophy

InterventionsPegcetacoplan

Overview

Phase: Not Applicable
Intervention: Pegcetacoplan
Conditions: Geographic Atrophy
Sponsor: Apellis Pharmaceuticals, Inc.
Enrollment: 300
Locations: 34
Primary Endpoint: Incidence of ocular AEs in treated eyes overall and according to physician-directed treatment interval based on US prescribing information
Status: Active, not recruiting
Last Updated: last month

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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

Registry

clinicaltrials.gov

Start Date

September 28, 2023

End Date

June 1, 2027

Last Updated

last month

Study Type

Observational

Sex

All

Investigators

Sponsor

Apellis Pharmaceuticals, Inc.

Responsible Party

Sponsor

Eligibility Criteria

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:
•Nonsubfoveal lesion(s)
•GA lesion visualized in its entirety on the macula-centered OCT image and not contiguous with any areas of peripapillary atrophy
•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

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

Arms & Interventions

Observation

Intervention: Pegcetacoplan

Outcomes

Primary Outcomes

Incidence of ocular AEs in treated eyes overall and according to physician-directed treatment interval based on US prescribing information

Time Frame: Up to 36 months

Incidence of other events of interest

Time Frame: Up to 36 months

Secondary Outcomes

If only one eye is treated, the criteria for determination of the eye to be treated(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 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)
The duration of persistence on treatment: continuous medication usage until a gap of ≥3 months(Up to 36 months)
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)
The treatment interval (mean days between injections) over time(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 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)