APL-3007 + Pegcetacoplan for Macular Degeneration

Recruiting at 1 trial location

Overseen By Apellis Clinical Trial Information Line

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Apellis Pharmaceuticals, Inc.

Must be taking: Syfovre

Disqualifiers: Uncontrolled diseases, Liver disease, Autoimmune disorders, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

240 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of a new treatment combining APL-3007 and pegcetacoplan for individuals with geographic atrophy, a condition causing vision loss due to age-related macular degeneration. Participants will receive different doses of APL-3007 or a placebo, alongside pegcetacoplan. The trial suits individuals aged 60 or older who have been diagnosed with geographic atrophy and have received Syfovre treatment for at least six months. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it does mention that participants should not have had certain treatments or conditions recently, so it's best to discuss your specific medications with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that both APL-3007 and pegcetacoplan are generally safe. APL-3007 has been tested for age-related macular degeneration, yielding positive results with no major side effects reported.

The FDA has already approved pegcetacoplan for other conditions like C3G and primary IC-MPGN, confirming its safety for people. In a previous study, most patients continued using pegcetacoplan, indicating it did not cause serious side effects.

Overall, past research has demonstrated good safety for both treatments. Prospective trial participants can feel reassured by this evidence. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about APL-3007 combined with pegcetacoplan for macular degeneration because it offers a fresh approach to tackling this eye condition. Unlike traditional treatments that primarily aim to slow down the progression of vision loss, APL-3007 introduces a new mechanism by specifically targeting key pathways involved in the disease process. Additionally, the use of pegcetacoplan, known for its role in complement inhibition, enhances the potential to reduce inflammation and damage in retinal cells. This combination not only aims to preserve vision but also potentially improves it, offering hope for better outcomes than current therapies.

What evidence suggests that this trial's treatments could be effective for macular degeneration?

Research has shown that pegcetacoplan effectively treats geographic atrophy, a type of age-related macular degeneration. Studies have found it can slow the growth of atrophy by up to 42%, offering promise for reducing vision loss.

In this trial, participants will receive different treatments to evaluate their effectiveness. Some participants will receive APL-3007 combined with pegcetacoplan, aiming to block certain harmful processes in the eye that lead to macular degeneration. Although data on APL-3007 is limited, researchers are carefully studying its combination with pegcetacoplan for potential benefits. Early results suggest this combination could provide a more comprehensive treatment. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

This trial is for people aged 60 or older with a specific eye condition called Geographic Atrophy due to Age-Related Macular Degeneration. They must have clear eyes, good enough vision, and have been treated with Syfovre regularly. Participants need vaccinations against certain diseases and agree to use contraception if applicable. Exclusions include allergies to the study drugs, substance abuse, recent live vaccines, certain health conditions, pregnancy or breastfeeding.

Inclusion Criteria

My eyes can be clearly imaged for the study.

I am 60 years old or older.

NL-BCVA of 50 letters or better using early treatment diabetic retinopathy study (ETDRS) charts (approximately 20/100 Snellen equivalent)

Exclusion Criteria

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

Evidence of ongoing drug or alcohol abuse or dependence

Intention to donate sperm during this study or within 90 days after the last dose of study drug

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multidose APL-3007 in combination with Syfovre/Pegcetacoplan (APL-2) or placebo

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• APL-2
• APL 3007

Trial Overview The study tests APL-3007 in combination with an existing treatment (Syfovre/Pegcetacoplan) for macular degeneration compared to a placebo. It's randomized and masked so neither participants nor researchers know who gets the real treatment versus placebo until after results are collected.

How Is the Trial Designed?

Group I: Group 2 (APL-3007 Dose/Frequency 2) Experimental Treatment 1 Intervention

APL-3007 + pegcetacoplan (APL-2)

Group II: Group 1 (APL-3007 Dose/Frequency 1 ) Experimental Treatment 1 Intervention

APL-3007 SC + pegcetacoplan (APL-2)

Group III: Group 3 (placebo) Placebo Group 1 Intervention

Placebo SC + pegcetacoplan (APL-2)

Who Is Running the Clinical Trial?

Apellis Pharmaceuticals, Inc.

Lead Sponsor

Trials

26

Recruited

4,300+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07215390

Study Details | NCT07215390 | A Phase 2, Randomized, ... A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of ...

2. investors.apellis.com investors.apellis.com/news-releases/news-release-details/apellis-pharmaceuticals-reports-second-quarter-2025-financial

Apellis Pharmaceuticals Reports Second Quarter 2025 ... Initiated the Phase 2 study of APL-3007 + SYFOVRE; potential next generation treatment aimed at comprehensively blocking complement activity in ...

3. theglobeandmail.com theglobeandmail.com/investing/markets/stocks/APLS/pressreleases/35744160/apellis-pharmaceuticals-promising-phase-2-study-on-geographic-atrophy-treatment/

Apellis Pharmaceuticals' Promising Phase 2 Study on ... The study aims to assess the effectiveness and safety of APL-3007, in combination with Syfovre/Pegcetacoplan, for treating geographic atrophy ...

4. synapse.patsnap.com synapse.patsnap.com/drug/38bd8a9e41da4cfa9304a8bec8ba8f7e

APL-3007 - Drug Targets, Indications, Patents 100 Clinical Results associated with APL-3007. Login to view more data. 100 Translational Medicine associated with APL-3007. Login to view more data.

5. sigma.larvol.com sigma.larvol.com/company.php?CompanyId=564224&tab=newstrac

Apellis News Our results show (similarly as other studies) that currently almost all PNH patients treated with complement inhibitors may survive 10 years or more with ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40280279/

Pegcetacoplan Treatment for Geographic Atrophy in Age- ... Results: Through the first 12 months of GALE, 92.0% (727/790) patient retention was observed. Across all eyes, including eyes with nonsubfoveal and subfoveal GA ...