Clinical trial NCT06161584: 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
Official title: 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 type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Geographic Atrophy
Lead sponsor: Apellis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-12-08
Enrollment: 300
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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


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:

  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

Primary outcomes:
measure: Incidence of ocular AEs in treated eyes overall and according to physician-directed treatment interval based on US prescribing information
timeFrame: Up to 36 months
measure: Incidence of other events of interest
timeFrame: Up to 36 months
Secondary outcomes:
measure: Assess real-world treatment patterns and use of pegcetacoplan considering the proportion of participants with bilateral GA treated with pegcetacoplan in both eyes
timeFrame: Up to 36 months
measure: If only one eye is treated, the criteria for determination of the eye to be treated
timeFrame: Up to 36 months
measure: The treatment interval (mean days between injections) over time
timeFrame: Up to 36 months
measure: 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
timeFrame: Up to 36 months
measure: The treatment adherence to planned dosing frequency: the number of injections administered divided by the number of injections planned
timeFrame: Up to 36 months
measure: The duration of persistence on treatment: continuous medication usage until a gap of ≥3 months
timeFrame: Up to 36 months
measure: The treatment discontinuation and/or switching to other agents approved for treatment of GA
timeFrame: Up to 36 months
measure: The management of new-onset exudative age-related macular degeneration (eAMD) events in eyes treated with pegcetacoplan in clinical practice
timeFrame: Up to 36 months
measure: The proportion of treated eyes that develop eAMD that receive anti-vascular endothelial growth factor (VEGF) injections
timeFrame: Up to 36 months
measure: The number of anti-VEGF injections per month from diagnosis of eAMD
timeFrame: Up to 36 months
measure: The proportion of anti-VEGF injections given on same day in the same eye as pegcetacoplan
timeFrame: Up to 36 months
Other outcomes:
Locations:
city: Bakersfield
country: United States
facility: California Retina Consultants (01-026)
state: California
city: Beverly Hills
country: United States
facility: Retina-Vitreous Associates Medical Group (01-020)
state: California
city: Fullerton
country: United States
facility: Illinois Retina Associates (01-035)
state: California
city: Modesto
country: United States
facility: Retinal Consultants Medical Group Inc (01-008)
state: California
city: Poway
country: United States
facility: Retina Consultants San Diego Inc. (01-018)
state: California
city: Santa Barbara
country: United States
facility: California Retina Consultants (01-027)
state: California
city: Walnut Creek
country: United States
facility: Bay Area Retina Associates (01-005)
state: California
city: Lakewood
country: United States
facility: Colorado Retina Associates, PLLC (01-011)
state: Colorado
city: Pensacola
country: United States
facility: Retina Specialty Institute (01-019)
state: Florida
city: Augusta
country: United States
facility: South East Retina Center, PC (01-030)
state: Georgia
city: Marietta
country: United States
facility: Georgia Retina (01-032)
state: Georgia
city: Oak Park
country: United States
facility: Illinois Retina Associates (01-037)
state: Illinois
city: Baltimore
country: United States
facility: The Retina Care Center (01-023)
state: Maryland
city: Hagerstown
country: United States
facility: Cumberland Valley Retina Consultants, P.C. (01-002)
state: Maryland
city: Hagerstown
country: United States
facility: Mid Atlantic Retina Specialist (01-029)
state: Maryland
city: Edina
country: United States
facility: Retina Consultants of Minnesota, PLLC (01-025)
state: Minnesota
city: Cherry Hill
country: United States
facility: Mid Atlantic Retina Research (01-014)
state: New Jersey
city: Cherry Hill
country: United States
facility: Mid Atlantic Retina (01-031)
state: New Jersey
city: Oceanside
country: United States
facility: OCLI Research Department (01-007)
state: New York
city: Westbury
country: United States
facility: Long Island Vitreoretinal Consultants (01-013)
state: New York
city: Asheville
country: United States
facility: Western Carolina Retinal Associates, PA (01-009)
state: North Carolina
city: Cleveland
country: United States
facility: Retina Associates of Cleveland, Inc. (01-017)
state: Ohio
city: Cleveland
country: United States
facility: Retina Associates of Cleveland, Inc. (01-022)
state: Ohio
city: Youngstown
country: United States
facility: Retina Associates of Cleveland, Inc. (01-016)
state: Ohio
city: Bethlehem
country: United States
facility: Mid Atlantic Retina (01-001)
state: Pennsylvania
city: York
country: United States
facility: The Retina Care Center (01-034) - Satellite Site
state: Pennsylvania
city: Rapid City
country: United States
facility: Black Hills Regional Eye Institute (01-010)
state: South Dakota
city: Nashville
country: United States
facility: Tennessee Retina (01-033)
state: Tennessee
city: Abilene
country: United States
facility: Integrated Clinical Research (01-024)
state: Texas
city: Bellaire
country: United States
facility: Retina Consultants of Texas (01-004)
state: Texas
city: Dallas
country: United States
facility: Texas Retina Associates (01-028)
state: Texas
city: The Woodlands
country: United States
facility: Retina Consultants of Texas (01-003)
state: Texas
city: Fairfax
country: United States
facility: The Retina Group of Washington (01-021)
state: Virginia
city: Bellevue
country: United States
facility: Pacific Northwest Retina (01-036)
state: Washington
Study officials:
References: