New drug combo aims to halt blindness from 'Dry' macular degeneration

NCT ID NCT07215390

Disease control Sponsor: Apellis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 2 trial tests whether adding APL-3007 to the existing drug Syfovre (pegcetacoplan) can better slow geographic atrophy, an advanced form of dry age-related macular degeneration that causes vision loss. About 240 adults aged 60 and older with the condition will receive either the combination or a placebo via eye injections. The study measures changes in retinal damage and safety over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: APL-3007 combined with pegcetacoplan (Syfovre)
What this could lead to: If successful, this combination could slow or stop the progression of geographic atrophy, a leading cause of blindness in older adults.
What could go wrong: This is an early Phase 2 trial with only 240 participants, so results may not confirm benefit. The treatment involves eye injections, which carry risks like infection or inflammation.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 240 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Nov 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

60 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

age-related macular degeneration

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Geographic atrophy secondary to age-related macular degeneration

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

78 sites. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

Advance Retina Institute
RECRUITING

Bonita Springs, Florida, 34134, United States
Advanced Vision Research Institute
RECRUITING

Longmont, Colorado, 80503, United States
Advancing Research International. LLC (01090)
RECRUITING

Indianapolis, Indiana, 46214, United States
Associated Retina Consultants (01065)
RECRUITING

Gilbert, Arizona, 85297, United States
Associated Retina Consultants - Phoenix
RECRUITING

Phoenix, Arizona, 85020, United States
Austin Retina Associate (01046)
RECRUITING

San Marcos, Texas, 78666, United States
Austin Retina Associates
RECRUITING

Austin, Texas, 78705, United States
Austin Retina Associates - Round Rock (01032)
RECRUITING

Round Rock, Texas, 78681, United States
Barnet Dulaney Perkins Eye Center (01018)
RECRUITING

Mesa, Arizona, 85206, United States
Bay Area Retina Associates - Walnut Creek
RECRUITING

Walnut Creek, California, 94598, United States
California Retina Consultants (01054)
RECRUITING

Oxnard, California, 93036, United States
California Retina Consultants (CRC) - Bakersfield Office
RECRUITING

Bakersfield, California, 93309, United States
Cascade Medical Research Institute, LLC (01070)
RECRUITING

Springfield, Oregon, 97477, United States
Center for Retina and Macular Disease (01026)
RECRUITING

Winter Haven, Florida, 33880, United States
Charleston Neuroscience Institute (01037)
RECRUITING

Beaufort, South Carolina, 29902, United States
Charleston Neuroscience Institute LLC - Ladson
RECRUITING

Ladson, South Carolina, 29456, United States
Charleston Neuroscience Institute, LLC - Mount Pleasant (01051)
RECRUITING

Mt. Pleasant, South Carolina, 29464, United States
ClearVista Clinical Research, LLC (01096)
RECRUITING

Bayonet Point, Florida, 34667, United States
Colorado Retina Associates, PLLC
RECRUITING

Lakewood, Colorado, 80228, United States
Connecticut Eye Consultants, PC (01074)
RECRUITING

Danbury, Connecticut, 06810, United States
Cumberland Valley Retina Consultants (CVRC)
RECRUITING

Hagerstown, Maryland, 21740, United States
Envision Ocular, LLC (01027)
RECRUITING

Bloomfield, New Jersey, 07003, United States
Eye Associates of Pinellas (01020)
RECRUITING

Pinellas Park, Florida, 33782, United States
Eye Care Specialists
RECRUITING

Kingston, Pennsylvania, 18704, United States
EyeHealth Northwest
RECRUITING

Portland, Oregon, 97225, United States
EyeHealth Northwest (01006)
RECRUITING

Portland, Oregon, 97228, United States
Florida Retina Institute (01053)
RECRUITING

Orlando, Florida, 32806, United States
Florida Retina Institute - Orlando (01053)
RECRUITING

Orlando, Florida, 32806, United States
Illinois Retina Associates, S.C. - Oak Park (01061)
RECRUITING

Oak Park, Illinois, 60304, United States
Long Island Vitreoretinal Consultants (01050)
RECRUITING

Great Neck, New York, 11021, United States
Long Island Vitreoretinal Consultants - Westbury
RECRUITING

Long Island City, New York, 11590, United States
Mid Atlantic Retina (01039)
RECRUITING

Cherry Hill, New Jersey, 08034, United States
Mid Atlantic Retina - Philadelphia (01017)
RECRUITING

Philadelphia, Pennsylvania, 19107, United States
Midwest Eye Institute (01066)
RECRUITING

Carmel, Indiana, 46032, United States
Mississippi Retina Associates
RECRUITING

Madison, Mississippi, 39110, United States
NJ Retina (01030)
RECRUITING

Toms River, New Jersey, 08755, United States
NJ Retina (01031)
RECRUITING

Edison, New Jersey, 08820, United States
Ophthalmology Ltd. (01084)
RECRUITING

Sioux Falls, South Dakota, 57108, United States
Orange County Retina Medical Group
RECRUITING

Santa Ana, California, 92705, United States
Piedmont Eye Center (01097)
RECRUITING

Lynchburg, Virginia, 24502, United States
Research Network Arizona
RECRUITING

Scottsdale, Arizona, 46214, United States
Retina Assocaites of Cleveland, Inc (01048)
RECRUITING

Cleveland, Ohio, 44122, United States
Retina Associates New Orleans - Jefferson Parish Location
RECRUITING

Metairie, Louisiana, 70001, United States
Retina Associates of Cleveland
RECRUITING

Cleveland, Ohio, 44130, United States
Retina Associates of Utah (01052)
RECRUITING

Salt Lake City, Utah, 84107, United States
Retina Center of Texas - Southlake
RECRUITING

Southlake, Texas, 76092, United States
Retina Consultants P.C.
RECRUITING

Indianapolis, Indiana, 46214, United States
Retina Consultants San Diego
RECRUITING

Poway, California, 92064, United States
Retina Consultants of Carolina, PA (01073)
RECRUITING

Greenville, South Carolina, 29605, United States
Retina Consultants of Orange County - Fullerton Office
RECRUITING

Fullerton, California, 93825, United States
Retina Consultants of Texas (01055)
RECRUITING

Schertz, Texas, 78154, United States
Retina Consultants of Texas - Abilene (01035)
RECRUITING

Abilene, Texas, 79606, United States
Retina Consultants of Texas - Bellaire (01067)
RECRUITING

Bellaire, Texas, 77401, United States
Retina Consultants of Texas - Katy
RECRUITING

Katy, Texas, 77494, United States
Retina Consultants of Texas - San Antonio Medical Center
RECRUITING

San Antonio, Texas, 78240, United States
Retina Consultants of Texas - The Woodlands
RECRUITING

The Woodlands, Texas, 73384, United States
Retina Macula Institute
RECRUITING

Torrance, California, 90503, United States
Retina Specialists
RECRUITING

Towson, Maryland, 21204, United States
Retina Specialty Institute - Pensacola
RECRUITING

Pensacola, Florida, 32503, United States
Retina Vitreous Associates of Florida
RECRUITING

Tampa, Florida, 33617, United States
Retina Vitreous Associates of Florida (RVA) - Saint Petersburg
RECRUITING

St. Petersburg, Florida, 33711, United States
Retinal Diagnostic Center
RECRUITING

Campbell, California, 95008, United States
Retinal Research Institute, LLC (01021)
RECRUITING

Phoenix, Arizona, 85053, United States
Salehi Retina Institute, Inc dba Retina Associate or Southern California (01034)
RECRUITING

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

Huntington Beach, California, 92647, United States
Salt Lake Retina (01044)
RECRUITING

West Jordan, Utah, 84088, United States
Southeast Retina Center
RECRUITING

Augusta, Georgia, 30909, United States
Tennessee Retina, PC (01063)
RECRUITING

Nashville, Tennessee, 37203, United States
Texas Retina Associates
RECRUITING

Dallas, Texas, 75231, United States
The Retina Care Center
RECRUITING

Baltimore, Maryland, 21209, United States
The Retina Group of Washington (01089)
RECRUITING

Chevy Chase, Maryland, 29902, United States
The Retina Partners - Encino
RECRUITING

Encino, California, 07003, United States
Tyler Retina Consultants
RECRUITING

Tyler, Texas, 75703, United States
University of Wisconsin Health - University Station Clinic
RECRUITING

Madison, Wisconsin, 53705, United States
Valley Retina Institute- McAllen (01076)
RECRUITING

McAllen, Texas, 78503, United States
Verum Research, LLC
RECRUITING

Eugene, Oregon, 97401, United States
Vision Research Center Eye Associates of New Mexico (01023)
RECRUITING

Albuquerque, New Mexico, 87109, United States
Vision Research Solutions
RECRUITING

Indianapolis, Indiana, 21044, United States

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