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Purpose

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

Condition

Eligibility

Eligible Ages
Over 60 Years
Eligible Sex
All
Accepts 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Group 1 (APL-3007 Dose/Frequency 1 ) 		APL-3007 SC + pegcetacoplan (APL-2)
  • Drug: APL-3007, pegcetacoplan (APL-2)
    Complement C3 inhibitor
Experimental
Group 2 (APL-3007 Dose/Frequency 2) 		APL-3007 + pegcetacoplan (APL-2)
  • Drug: APL-3007, pegcetacoplan (APL-2)
    Complement C3 Inhibitor
Placebo Comparator
Group 3 (placebo) 		Placebo SC + pegcetacoplan (APL-2)
  • Other: Placebo, Syfovre
    Complement C3 Inhibitor

Recruiting Locations

Associated Retina Consultants (01065)
Gilbert, Arizona 85297

Barnet Dulaney Perkins Eye Center (01018)
Mesa, Arizona 85206

Associated Retina Consultants - Phoenix
Phoenix, Arizona 85020

Retinal Research Institute, LLC (01021)
Phoenix, Arizona 85053

Research Network Arizona
Scottsdale, Arizona 46214

California Retina Consultants (CRC) - Bakersfield Office
Bakersfield, California 93309

Retinal Diagnostic Center
Campbell, California 95008

The Retina Partners - Encino
Encino, California 07003

Retina Consultants of Orange County - Fullerton Office
Fullerton, California 93825

Salehi Retina Institute, Inc dba Retina Associate or Southern California (01034)
Huntington Beach, California 92647

Salehi Retina Institute, Inc. Dba Retina Associates of Southern California (01034)
Huntington Beach, California 92647

California Retina Consultants (01054)
Oxnard, California 93036

Retina Consultants San Diego
Poway, California 92064

Orange County Retina Medical Group
Santa Ana, California 92705

Retina Macula Institute
Torrance, California 90503

Bay Area Retina Associates - Walnut Creek
Walnut Creek, California 94598

Colorado Retina Associates, PLLC
Lakewood, Colorado 80228

Advanced Vision Research Institute
Longmont, Colorado 80503

Connecticut Eye Consultants, PC (01074)
Danbury, Connecticut 06810

ClearVista Clinical Research, LLC (01096)
Bayonet Point, Florida 34667

Advance Retina Institute
Bonita Springs, Florida 34134

Florida Retina Institute (01053)
Orlando, Florida 32806

Florida Retina Institute - Orlando (01053)
Orlando, Florida 32806

Retina Specialty Institute - Pensacola
Pensacola, Florida 32503

Eye Associates of Pinellas (01020)
Pinellas Park, Florida 33782

Retina Vitreous Associates of Florida (RVA) - Saint Petersburg
St. Petersburg, Florida 33711

Retina Vitreous Associates of Florida
Tampa, Florida 33617

Center for Retina and Macular Disease (01026)
Winter Haven, Florida 33880

Southeast Retina Center
Augusta, Georgia 30909

Illinois Retina Associates, S.C. - Oak Park (01061)
Oak Park, Illinois 60304

Midwest Eye Institute (01066)
Carmel, Indiana 46032

Vision Research Solutions
Indianapolis, Indiana 21044

Advancing Research International. LLC (01090)
Indianapolis, Indiana 46214

Retina Consultants P.C.
Indianapolis, Indiana 46214

Retina Associates New Orleans - Jefferson Parish Location
Metairie, Louisiana 70001

The Retina Care Center
Baltimore, Maryland 21209

The Retina Group of Washington (01089)
Chevy Chase, Maryland 29902

Cumberland Valley Retina Consultants (CVRC)
Hagerstown, Maryland 21740

Retina Specialists
Towson, Maryland 21204

Mississippi Retina Associates
Madison, Mississippi 39110

Envision Ocular, LLC (01027)
Bloomfield, New Jersey 07003

Mid Atlantic Retina (01039)
Cherry Hill, New Jersey 08034

NJ Retina (01031)
Edison, New Jersey 08820

NJ Retina (01030)
Toms River, New Jersey 08755

Vision Research Center Eye Associates of New Mexico (01023)
Albuquerque, New Mexico 87109

Long Island Vitreoretinal Consultants (01050)
Great Neck, New York 11021

Long Island Vitreoretinal Consultants - Westbury
Long Island City, New York 11590

Retina Assocaites of Cleveland, Inc (01048)
Cleveland, Ohio 44122

Retina Associates of Cleveland
Cleveland, Ohio 44130

Verum Research, LLC
Eugene, Oregon 97401

EyeHealth Northwest
Portland, Oregon 97225

EyeHealth Northwest (01006)
Portland, Oregon 97228

Cascade Medical Research Institute, LLC (01070)
Springfield, Oregon 97477

Eye Care Specialists
Kingston, Pennsylvania 18704

Mid Atlantic Retina - Philadelphia (01017)
Philadelphia, Pennsylvania 19107

Charleston Neuroscience Institute (01037)
Beaufort, South Carolina 29902

Retina Consultants of Carolina, PA (01073)
Greenville, South Carolina 29605

Charleston Neuroscience Institute LLC - Ladson
Ladson, South Carolina 29456

Charleston Neuroscience Institute, LLC - Mount Pleasant (01051)
Mt. Pleasant, South Carolina 29464

Ophthalmology Ltd. (01084)
Sioux Falls, South Dakota 57108

Tennessee Retina, PC (01063)
Nashville, Tennessee 37203

Retina Consultants of Texas - Abilene (01035)
Abilene, Texas 79606

Austin Retina Associates
Austin, Texas 78705

Retina Consultants of Texas - Bellaire (01067)
Bellaire, Texas 77401

Texas Retina Associates
Dallas, Texas 75231

Retina Consultants of Texas - Katy
Katy, Texas 77494

Valley Retina Institute- McAllen (01076)
McAllen, Texas 78503

Austin Retina Associates - Round Rock (01032)
Round Rock, Texas 78681

Retina Consultants of Texas - San Antonio Medical Center
San Antonio, Texas 78240

Austin Retina Associate (01046)
San Marcos, Texas 78666

Retina Consultants of Texas (01055)
Schertz, Texas 78154

Retina Center of Texas - Southlake
Southlake, Texas 76092

Retina Consultants of Texas - The Woodlands
The Woodlands, Texas 73384

Tyler Retina Consultants
Tyler, Texas 75703

Retina Associates of Utah (01052)
Salt Lake City, Utah 84107

Salt Lake Retina (01044)
West Jordan, Utah 84088

Piedmont Eye Center (01097)
Lynchburg, Virginia 24502

University of Wisconsin Health - University Station Clinic
Madison, Wisconsin 53705

More Details

Status
Recruiting
Sponsor
Apellis Pharmaceuticals, Inc.

Study Contact

Apellis Clinical Trial Information Line
617-977-5700
clinicaltrials@apellis.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.