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Clinical Trials in the USA / NCT07215390
Recruiting Phase 2

A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio

NCT07215390 · tracked via the Priya Life Science USA tracker
Sponsor
Apellis Pharmaceuticals, Inc.
Phase
Phase 2
Started
2025-06-23
Last updated
2026-06-24

Condition(s) studied

Geographic Atrophy Secondary to Age-related Macular Degeneration

Investigational drug(s) / intervention(s)

APL-3007, pegcetacoplan (APL-2)APL-3007, pegcetacoplan (APL-2)Placebo, Syfovre

APL-3007, pegcetacoplan (APL-2): Complement C3 inhibitor

APL-3007, pegcetacoplan (APL-2): Complement C3 Inhibitor

Placebo, Syfovre: Complement C3 Inhibitor

Study summary

A Phase 2, Randomized, Placebo-controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL-3007 in Combination with Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed with Geographic Atrophy Secondary to Age-Related Macular Degeneration

Eligibility

Sex
ALL
Min age
60 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * The study eye must meet all inclusion criteria. If both eyes meet the inclusion criteria, the eye with better normal luminance visual acuity at the screening visit will be designated as the study eye. If both eyes have the same visual acuity, the right eye will be used as the study eye. * Aged ≥60 years * Clinical diagnosis of GA of the macula secondary to AMD in one or both eyes, as determined by the investigator and confirmed by the reading center * NL-BCVA of 50 letters or better using early treatment diabetic retinopathy study (ETDRS) charts (approximately 20/100 Snellen equivalent) * Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images in the study eye as determined by the investigator * Prior treatment for GA in the study eye using Syfovre at 6-8 weeks interval for at least 6 months but no more than 24 months. Participants will be included if the participant has had at least 2 Syfovre injections in the last 6 months before screening. * The GA lesion in at least 1 eye (designated as the study eye) must meet the following criteria as determined by the central reading center's OCT based RPE assessment of imaging at screening: * Total GA area must be ≥2.5 and ≤17.5 mm2 (1-7 disk areas \[DA\]) * If GA is multifocal, at least 1 focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 7a * The entire GA lesion must be completely visualized in the field of view of the OCT, with the GA RPE lesion border \>500 µm from the edge of the imaging window or any areas of peripapillary atrophy. * Nonsubfoveal lesion with border of GA lesion not encroaching center of the fovea. Distance of GA RPE lesion from center of the fovea \>0 based on OCT imaging. * Presence of any pattern of hyperautofluorescence based on FAF imaging in the junctional zone of GA. Absence of hyperautofluorescence (ie, pattern = none) is exclusionary. * Documented evidence of vaccination within 5 years prior to screening, or willing to initiate vaccinations at least 14 days prior to dosing against: * Streptococcus pneumoniae (with a pneumococcal conjugate vaccine 15 \[PCV15\] or 20 \[PCV20\] or with pneumococcal polysaccharide vaccine 23 \[PPSV23\]), * Haemophilus influenzae (type B) (with Hib vaccine), * Neisseria meningitidis types A, C, W, and Y (with a quadrivalent meningococcal conjugate vaccine \[eg, Menactra or MenQuadfi\]), and * Neisseria meningitidis type B (with a meningococcal serogroup B vaccine \[eg, Bexsero\]) Female participants must be: * Women of non-childbearing potential (WONCBP), or * Women of childbearing 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 (see Section 10.9.5.1) Male participants must be surgically sterile or must agree to use highly effective contraception from screening through the duration of the study Willing and able to provide informed consent and adhere to the study visit schedule and other protocol requirements Exclusion Criteria: 1. Uncontrolled, clinically relevant history of any gastrointestinal, renal, hepatic, bronchopulmonary, neurological, psychiatric, cardiovascular, endocrinological, hematological or allergic disease, metabolic disorder, or cancer 2. History or presence of hepatic cirrhosis or other liver disease that may increase the risk of drug-induced liver injury 3. History or presence of systemic autoimmune disorders, with the exception of well controlled Hashimoto's thyroiditis 4. History of allergy, hypersensitivity, or serious adverse reaction to siRNA therapy or related compounds, or allergy to any of the components of the study drug 5. Clinically meaningful abnormalities on diagnostic and laboratory testing must be adjudicated by the sponsor's medical monitor and include: 1. Cardiac Sustained resting heart rate outside of range of 40 to 100 beats/minute, or a heart rhythm that is not sinus rhythm, confirmed on repeat testing within a maximum of 30 minutes, at screening History or evidence of hereditary short QT syndrome Fridericia's corrected QT interval (QTcF) \>480 milliseconds, or the PR interval outside the range of 120 to 220 milliseconds, confirmed on repeat testing within a maximum of 30 minutes at screening Any clinically relevant features of acute coronary syndrome (unstable angina, myocardial infarction) Any other ECG parameters outside of age-adjusted normative range 2. Hepatic AST or ALT \>1.3 × ULN Total bilirubin \>1.1 × ULN Any 2 LFTs \>1.1 × ULN Any other LFT parameters outside of age-adjusted normative range 3. Renal Estimated glomerular filtration rate of less than \<45 mL/min/m2 as calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKD- EPI) creatinine equation for adults Any other renal function parameters outside of age-adjusted normative range 6. History or presence of malignancy (except history of basal or squamous cell carcinoma of the skin) that has not been successfully treated ≥1 year prior to enrollment 7. History or presence of recurrent or unexplained infections, HIV infection, hepatitis B, hepatitis C, or meningococcal infection 8. Participants with fever (defined as temperature \>100.4 °F/38 °C) or any acute infection (including COVID-19 infection or infection requiring antibiotic treatment) within 30 days of screening until dosing 9. Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator 10. Intention to donate sperm during this study or within 90 days after the last dose of study drug 11. Prior administration of APL-3007 12. Participation in an investigational product or medical device study within 5 half-lives of the investigational product before the screening visit 13. Has poor peripheral venous access or any abnormal skin conditions or potentially obscuring tattoos, pigmentation, or lesions in the area intended for SC injection that would interfere with SC injections or assessments of injection local tolerability 14. Any condition which could interfere with, or the treatment for which might interfere with, the conduct of the study or which would, in the opinion of the investigator or medical monitor(s), unacceptably increase the participant's risk by participating in the study 15. Has received a live vaccination (excluding seasonal flu vaccination) within 30 days prior to the first dose administration 16. Women who are pregnant or breastfeeding 17. Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the treatment period unlikely, or would make the participant an unsafe study candidate Ocular specific exclusion criteria apply to the study eye only, unless otherwise specified. 18. GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye 19. Any history of CNV (active or nonexudative/quiescent) in the study eye associated with AMD or any other cause, including any evidence of RPE rips, double layer sign, or evidence of neovascularization anywhere based on SD-OCT imaging and fluorescein angiography as assessed by the reading center 20. Presence of an active ocular disease that, in the opinion of the investigator, compromises or confounds visual function, including but not limited to, uveitis, other macular diseases (eg, clinically significant epiretinal membrane, full thickness macular hole or uncontrolled glaucoma/ocular hypertension); benign conditions in the opinion of the investigator such as peripheral retina dystrophy are not exclusionary. 21. History of IVT injection in the study eye within 3 months prior to the screening visit, with the exception of IVT Syfovre 22. History of any previous investigational product for GA treatment in either eye within 3 months (or 5 half-lives of the investigational drug, whichever is longer) prior to the screening visit 23. History of laser therapy in the macular region 24. 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

Primary outcome measure(s)

Trial sites (78)

FacilityCityRegionStatus
Associated Retina Consultants (01065) Gilbert Arizona Recruiting
Barnet Dulaney Perkins Eye Center (01018) Mesa Arizona Recruiting
Associated Retina Consultants - Phoenix Phoenix Arizona Recruiting
Retinal Research Institute, LLC (01021) Phoenix Arizona Recruiting
Research Network Arizona Scottsdale Arizona Recruiting
California Retina Consultants (CRC) - Bakersfield Office Bakersfield California Recruiting
Retinal Diagnostic Center Campbell California Recruiting
The Retina Partners - Encino Encino California Recruiting
Retina Consultants of Orange County - Fullerton Office Fullerton California Recruiting
Salehi Retina Institute, Inc dba Retina Associate or Southern California (01034) Huntington Beach California Recruiting
Salehi Retina Institute, Inc. Dba Retina Associates of Southern California (01034) Huntington Beach California Recruiting
California Retina Consultants (01054) Oxnard California Recruiting
Retina Consultants San Diego Poway California Recruiting
Orange County Retina Medical Group Santa Ana California Recruiting
Retina Macula Institute Torrance California Recruiting
Bay Area Retina Associates - Walnut Creek Walnut Creek California Recruiting
Colorado Retina Associates, PLLC Lakewood Colorado Recruiting
Advanced Vision Research Institute Longmont Colorado Recruiting
Connecticut Eye Consultants, PC (01074) Danbury Connecticut Recruiting
ClearVista Clinical Research, LLC (01096) Bayonet Point Florida Recruiting
Advance Retina Institute Bonita Springs Florida Recruiting
Florida Retina Institute (01053) Orlando Florida Recruiting
Florida Retina Institute - Orlando (01053) Orlando Florida Recruiting
Retina Specialty Institute - Pensacola Pensacola Florida Recruiting
Eye Associates of Pinellas (01020) Pinellas Park Florida Recruiting
Retina Vitreous Associates of Florida (RVA) - Saint Petersburg St. Petersburg Florida Recruiting
Retina Vitreous Associates of Florida Tampa Florida Recruiting
Center for Retina and Macular Disease (01026) Winter Haven Florida Recruiting
Southeast Retina Center Augusta Georgia Recruiting
Illinois Retina Associates, S.C. - Oak Park (01061) Oak Park Illinois Recruiting
Midwest Eye Institute (01066) Carmel Indiana Recruiting
Vision Research Solutions Indianapolis Indiana Recruiting
Advancing Research International. LLC (01090) Indianapolis Indiana Recruiting
Retina Consultants P.C. Indianapolis Indiana Recruiting
Retina Associates New Orleans - Jefferson Parish Location Metairie Louisiana Recruiting
The Retina Care Center Baltimore Maryland Recruiting
The Retina Group of Washington (01089) Chevy Chase Maryland Recruiting
Cumberland Valley Retina Consultants (CVRC) Hagerstown Maryland Recruiting
Retina Specialists Towson Maryland Recruiting
Mississippi Retina Associates Madison Mississippi Recruiting

+ 38 more sites — see the full list on the official registry below.

More Apellis Pharmaceuticals, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07215390 on ClinicalTrials.gov ↗ ← All trials in the USA