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 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: APL3007-GA-201
• Study Type: interventional
• Target: 240
• Locations: 1 country
• Sites: 43

Brief 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

Conditions

Geographic Atrophy Secondary to Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

• Change from baseline (CFB) in the area of artificial intelligence (AI)-based SD-OCT assessment of RPE lesion in the study eye

  At month 12

Secondary Outcomes (9)

• CFB in the area of AI-based OCT assessment of photoreceptor degeneration in the study eye

  At month 12

• Safety & tolerability based on adverse event (AE) reporting

  At month 12

• Safety as assessed by Best Corrected Visual Acuity (BCVA)

  At Month 12

• Change from baseline in Serum C3 level

  At Month 12

• CFB in RPE lesion area as assessed by FAF in the study eye

  At month 12

Study Arms (3)

Group 1 (APL-3007 Dose/Frequency 1 )

EXPERIMENTAL

APL-3007 SC + pegcetacoplan (APL-2)

Drug: APL-3007, pegcetacoplan (APL-2)

Group 2 (APL-3007 Dose/Frequency 2)

EXPERIMENTAL

APL-3007 + pegcetacoplan (APL-2)

Drug: APL-3007, pegcetacoplan (APL-2)

Group 3 (placebo)

PLACEBO COMPARATOR

Placebo SC + pegcetacoplan (APL-2)

Other: Placebo, Syfovre

Interventions

APL-3007, pegcetacoplan (APL-2) DRUG

Complement C3 inhibitor

Group 1 (APL-3007 Dose/Frequency 1 )

Placebo, Syfovre OTHER

Complement C3 Inhibitor

Group 3 (placebo)

Eligibility Criteria

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

You may qualify if:

• 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.
• 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 \[eg, Bexsero\])

You may not qualify if:

• Uncontrolled, clinically relevant history of any gastrointestinal, renal, hepatic, bronchopulmonary, neurological, psychiatric, cardiovascular, endocrinological, hematological or allergic disease, metabolic disorder, or cancer
• History or presence of hepatic cirrhosis or other liver disease that may increase the risk of drug-induced liver injury
• History or presence of systemic autoimmune disorders, with the exception of well controlled Hashimoto's thyroiditis
• 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
• Clinically meaningful abnormalities on diagnostic and laboratory testing must be adjudicated by the sponsor's medical monitor and include:
• 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
• Hepatic
• AST or ALT \>1.3 × ULN
• Total bilirubin \>1.1 × ULN
• Any 2 LFTs \>1.1 × ULN

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (43)

Barnet Dulaney Perkins Eye Center (01018)

Mesa, Arizona, 85206, United States

RECRUITING

Associated Retina Consultants - Phoenix

Phoenix, Arizona, 85020, United States

RECRUITING

Retinal Research Institute, LLC (01021)

Phoenix, Arizona, 85053, United States

RECRUITING

California Retina Consultants (CRC) - Bakersfield Office

Bakersfield, California, 93309, United States

RECRUITING

The Retina Partners - Encino

Encino, California, 07003, United States

RECRUITING

Retina Consultants of Orange County - Fullerton Office

Fullerton, California, 93825, United States

RECRUITING

Salehi Retina Institute, Inc dba Retina Associate or Southern California (01034)

Huntington Beach, California, 92647, United States

RECRUITING

Retina Consultants San Diego

Poway, California, 92064, United States

RECRUITING

Orange County Retina Medical Group

Santa Ana, California, 92705, United States

RECRUITING

Bay Area Retina Associates - Walnut Creek

Walnut Creek, California, 94598, United States

RECRUITING

Colorado Retina Associates, PLLC

Lakewood, Colorado, 80228, United States

RECRUITING

Advanced Vision Research Institute

Longmont, Colorado, 80503, United States

RECRUITING

Florida Retina Institute - Orlando (01053)

Orlando, Florida, 32806, United States

RECRUITING

Retina Specialty Institute - Pensacola

Pensacola, Florida, 32503, United States

RECRUITING

Retina Vitreous Associates of Florida (RVA) - Saint Petersburg

St. Petersburg, Florida, 33711, United States

RECRUITING

Center for Retina and Macular Disease (01026)

Winter Haven, Florida, 33880, United States

RECRUITING

Southeast Retina Center

Augusta, Georgia, 30909, United States

RECRUITING

Illinois Retina Associates, S.C. - Oak Park (01061)

Oak Park, Illinois, 60304, United States

RECRUITING

Retina Consultants P.C.

Indianapolis, Indiana, 46214, United States

RECRUITING

Retina Associates New Orleans - Jefferson Parish Location

Metairie, Louisiana, 70001, United States

RECRUITING

Cumberland Valley Retina Consultants (CVRC)

Hagerstown, Maryland, 21740, United States

RECRUITING

Mississippi Retina Associates

Madison, Mississippi, 39110, United States

RECRUITING

Long Island Vitreoretinal Consultants - Westbury

Long Island City, New York, 11590, United States

RECRUITING

Retina Assocaites of Cleveland, Inc (01048)

Cleveland, Ohio, 44122, United States

RECRUITING

Verum Research, LLC

Eugene, Oregon, 97401, United States

RECRUITING

EyeHealth Northwest

Portland, Oregon, 97225, United States

RECRUITING

Mid Atlantic Retina - Philadelphia (01017)

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Charleston Neuroscience Institute LLC - Ladson

Ladson, South Carolina, 29456, United States

RECRUITING

Charleston Neuroscience Institute, LLC - Mount Pleasant (01051)

Mt. Pleasant, South Carolina, 29464, United States

RECRUITING

Retina Consultants of Texas - Abilene (01035)

Abilene, Texas, 79606, United States

RECRUITING

Austin Retina Associates

Austin, Texas, 78705, United States

RECRUITING

Retina Consultants of Texas - Bellaire (01067)

Bellaire, Texas, 77401, United States

RECRUITING

Texas Retina Associates

Dallas, Texas, 75231, United States

RECRUITING

Retina Consultants of Texas - Katy

Katy, Texas, 77494, United States

RECRUITING

Valley Retina Institute- McAllen (01076)

McAllen, Texas, 78503, United States

RECRUITING

Austin Retina Associates - Round Rock (01032)

Round Rock, Texas, 78681, United States

RECRUITING

Retina Consultants of Texas - San Antonio Medical Center

San Antonio, Texas, 78240, United States

RECRUITING

Austin Retina Associate (01046)

San Marcos, Texas, 78666, United States

RECRUITING

Retina Consultants of Texas (01055)

Schertz, Texas, 78154, United States

RECRUITING

Retina Center of Texas - Southlake

Southlake, Texas, 76092, United States

RECRUITING

Retina Consultants of Texas - The Woodlands

The Woodlands, Texas, 73384, United States

RECRUITING

Tyler Retina Consultants

Tyler, Texas, 75703, United States

RECRUITING

University of Wisconsin Health - University Station Clinic

Madison, Wisconsin, 53705, United States

RECRUITING

MeSH Terms

Interventions

pegcetacoplan

Central Study Contacts

Apellis Clinical Trial Information Line

CONTACT

617-977-5700; clinicaltrials@apellis.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2025
• First Posted: October 10, 2025
• Study Start: June 23, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): November 1, 2027
• Last Updated: March 30, 2026

Record last verified: 2025-10

Locations

US (43)