Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy

NCT03777332 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: APL2-103
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 5

Brief Summary

Safety Assessment of APL-2 in Patients with Geographic Atrophy

Conditions

Geographic Atrophy

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs)

  25 Months

Study Arms (3)

(Cohort 1) Pegcetacoplan, 15 mg/100 μL, monthly for up to 60 months

EXPERIMENTAL

Drug: PEGCETACOPLAN (APL-2)

(Cohort 2) Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months

EXPERIMENTAL

Drug: PEGCETACOPLAN (APL-2)

(Cohort 2) Pegcetacoplan, 15 mg/100 μL, every other month for up to 36 months

EXPERIMENTAL

Drug: PEGCETACOPLAN (APL-2)

Interventions

PEGCETACOPLAN (APL-2) DRUG

Complement (C3) Inhibitor

(Cohort 1) Pegcetacoplan, 15 mg/100 μL, monthly for up to 60 months; (Cohort 2) Pegcetacoplan, 15 mg/100 μL, every other month for up to 36 months; (Cohort 2) Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 60 years.
• Normal Luminance best corrected visual acuity of between 35 and 5 letters using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately between 20/200 and 20/800 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:
• Total GA area must be ≥ 2.5 (1 disk areas \[DA\]).
• 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.
• 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.

You may not qualify if:

• 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.
• Intraocular surgery (including lens replacement surgery) within 3 months prior to enrollment.
• History of laser therapy in the macular region.
• History of intraocular injection within 3 months prior to the screening visit.
• 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.
• Medical or psychiatric conditions that, in the opinion of the Investigator, make consistent follow-up over the 60-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 pegcetacoplan or any of the excipients in pegcetacoplan solution.
• Participated in the APL2-303 (Derby, NCT03525613) or APL2-304 (Oaks, NCT03525600) study with completion of the Month 24 visit and have been invited to participate in this study.
• a. Subjects who did not discontinue treatment but missed the Month 24 visit are also eligible to participate in this study.
• 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:

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (5)

California Retina Consultants

Bakersfield, California, 93309, United States

Location

Retina Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

The Retina Partners

Encino, California, 91436, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

Retina Consultants of Houston

The Woodlands, Texas, 77384, United States

Location

Related Publications (1)

• 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.

  PMID: 40280279 DERIVED

MeSH Terms

Conditions

Geographic Atrophy

Interventions

pegcetacoplan

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2018
• First Posted: December 17, 2018
• Study Start: November 5, 2018
• Primary Completion: April 1, 2021
• Study Completion: April 1, 2021
• Last Updated: November 10, 2021

Record last verified: 2021-11

Locations

US (5)