Pegcetacoplan for Age-Related Macular Degeneration

(GALE Trial)

No longer recruiting at 182 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Apellis Pharmaceuticals, Inc.

Disqualifiers: Eye diseases, Eye inflammation, Allergies, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 36 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores pegcetacoplan, an experimental treatment for individuals with geographic atrophy, a vision-affecting condition related to age-related macular degeneration (AMD). The trial aims to assess the long-term safety and effectiveness of this treatment. Participants will receive the treatment either monthly or every other month for up to three years. Suitable candidates have previously participated in specific studies on pegcetacoplan and have a clear view of the inside of their eyes for proper examination. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that pegcetacoplan is likely to be safe for humans?

Research has shown that pegcetacoplan is generally well-tolerated by people with age-related macular degeneration (AMD). In studies, some individuals experienced side effects, such as exudative AMD in 4.5% of cases and mild to moderate inflammation inside the eye in 1.9% of cases. These side effects are relatively uncommon.

Importantly, pegcetacoplan has undergone long-term safety studies, with up to four years of safety data available. This extensive research provides a clear understanding of what to expect from this treatment. Overall, while some risks exist, many people use pegcetacoplan without significant problems. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for AMD?

Researchers are excited about pegcetacoplan for age-related macular degeneration because it offers a novel approach compared to standard treatments like anti-VEGF injections. Unlike these existing options, which primarily focus on inhibiting vascular growth, pegcetacoplan targets a different pathway by inhibiting the complement system, which is believed to play a role in the progression of this disease. This new mechanism of action could potentially slow down vision loss more effectively. Additionally, pegcetacoplan provides flexible dosing schedules, with options for monthly or every-other-month injections, which might offer greater convenience and comfort for patients.

What evidence suggests that pegcetacoplan might be an effective treatment for age-related macular degeneration?

Research has shown that pegcetacoplan effectively treats geographic atrophy (GA) related to age-related macular degeneration (AMD). One study found that after 36 months, pegcetacoplan reduced GA growth by up to 42% in affected eyes. Another study observed a 37% reduction in GA growth rate after just one year of treatment. In this trial, participants will receive injections of pegcetacoplan either monthly or every other month, both of which have demonstrated increasing effectiveness over time. These findings suggest that pegcetacoplan can slow the progression of GA in AMD patients, making it a promising treatment option. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for people with a specific eye condition called geographic atrophy due to age-related macular degeneration, who were in previous related studies. They must have clear eyes for imaging and agree to birth control measures if applicable. Excluded are those with other significant eye diseases, current pregnancy or breastfeeding, known allergies to study substances, or conditions preventing study participation.

Inclusion Criteria

For subjects who participated in either the 303 or 304 studies, the condition listed below must also be met for participating in this study.

I am a woman.

Women who can have children must have a negative result of a blood pregnancy test on the first day of the study visit and agree to use ways to avoid pregnancy during the study and 90 days after the last dose of the study medication.

Exclusion Criteria

My eye does not have any disqualifying conditions for the study.

If, according to your doctor, any current disease that could directly interfere in your participation in the study or that could make it difficult for you to come to the scheduled visits during the next three years over the next 36 months.

If you currently are pregnant, breastfeed or have a positive pregnancy test.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravitreal pegcetacoplan (15 mg/100 μL) monthly or every other month for up to 36 months

36 months

Monthly or every other month visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive treatment to evaluate long-term safety and efficacy

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Pegcetacoplan (APL-2)

Trial Overview The long-term safety and effectiveness of Pegcetacoplan (APL-2) are being tested in this phase 3 extension study on individuals with geographic atrophy secondary to AMD who participated in earlier related trials.

How Is the Trial Designed?

Group I: Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months Experimental Treatment 1 Intervention

Participants from Study APL2-103 (NCT03777332) or those who completed the treatment at Month 24 from either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600) and were administered monthly intravitreal (IVT) pegcetacoplan (15 mg/100 μL) or monthly sham will receive IVT pegcetacoplan (15 mg/100 μL) monthly for up to approximately 36 months.

Group II: Pegcetacoplan, 15 mg/100 μL, every other month (EOM) for up to 36 months Experimental Treatment 1 Intervention

Participants from Study APL2-103 (NCT03777332) or those who completed the treatment at Month 24 from either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600) and were administered every other month (EOM) intravitreal (IVT) pegcetacoplan (15 mg/100 μL) or EOM sham will receive IVT pegcetacoplan (15 mg/100 μL) EOM for up to approximately 36 months.

Who Is Running the Clinical Trial?

Apellis Pharmaceuticals, Inc.

Lead Sponsor

Trials

26

Recruited

4,300+

Published Research Related to This Trial

In a study of 75 Japanese patients with neovascular age-related macular degeneration (AMD), pegaptanib sodium as maintenance therapy showed stable visual acuity over 54 weeks after successful induction therapy, with no significant changes in vision during this period.

The treatment was well-tolerated, with only 5.3% of patients reporting adverse events, and mean intraocular pressure remained stable, indicating a favorable safety profile for pegaptanib maintenance therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Maintenance therapy with pegaptanib sodium for neovascular age-related macular degeneration: an exploratory study in Japanese patients (LEVEL-J study). Ishibashi, T.[2022]

Abicipar pegol showed comparable efficacy in improving visual acuity and reducing retinal thickness in treatment-naïve patients with neovascular age-related macular degeneration (nAMD) in both Japan and the United States, with mean visual acuity changes of +7.8 to +8.9 letters in Japan and +4.4 to +10.1 letters in the U.S. after 16 weeks.

The treatment was generally safe, with only three cases of uveitis or vitritis reported among abicipar-treated patients, indicating a favorable safety profile for this medication.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of Abicipar Pegol (an Anti-VEGF DARPin Therapeutic) in Patients With Neovascular Age-Related Macular Degeneration: Studies in Japan and the United States. Kunimoto, D., Ohji, M., Maturi, RK., et al.[2019]

In a study of 253 patients with neovascular age-related macular degeneration (NV-AMD) treated with pegaptanib sodium, over 90% of patients experienced less than 15 letters of vision loss after 24 and 54 weeks, which is significantly better than the 70% reported in the VISION trial.

Pegaptanib was well tolerated among patients, with no serious adverse effects such as endophthalmitis or retinal detachment reported, indicating a favorable safety profile for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravitreal pegaptanib sodium for choroidal neovascularisation secondary to age-related macular degeneration: Pan-European experience. Sivaprasad, S., Hykin, P., Saeed, A., et al.[2016]

Citations

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

Pegcetacoplan Treatment for Geographic Atrophy in Age- ... Over 36 months, pegcetacoplan continued to reduce GA growth with increasing efficacy over time and reduced formation of new scotomatous points.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02503332

NCT02503332 | Study of Pegcetacoplan (APL-2) Therapy ... A single dose of 15 mg pegcetacoplan/100 µL will be administered via intravitreal injection in this study. Subjects will receive an injection every other month ...

3. investors.apellis.com investors.apellis.com/news-releases/news-release-details/phase-2-study-published-ophthalmology-highlights-efficacy-and

Phase 2 Study Published in Ophthalmology Highlights ... APL-2 was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S000293942500193X

Pegcetacoplan Treatment for Geographic Atrophy in Age ... Over 36 months, pegcetacoplan continued to reduce GA growth with increasing efficacy over time and reduced formation of new scotomatous points.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12534827/

Efficacy of Intravitreal Pegcetacoplan vs Avacincaptad Pegol ... Purpose: To evaluate the efficacy of intravitreal (IVT) pegcetacoplan monthly vs avacincaptad pegol monthly (primary analysis), and ...

6. syfovreecp.com syfovreecp.com/safety/

SYFOVRE® (pegcetacoplan injection) safety SYFOVRE has 4 years of long-term safety data1,2. Through 2 years ... age-related macular degeneration (AMD). IMPORTANT SAFETY INFORMATION. CONTRAINDICATIONS.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT02461771

Study Details | NCT02461771 | Assessment of Safety, ... The objective of this study is to provide initial safety, tolerability and pharmacokinetics information of intravitreal administration of pegcetacoplan in ...

8. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0002939425001990

Ocular Adverse Events Associated with Pegcetacoplan and ... These findings aim to enhance clinicians' understanding of the safety profiles of these agents, enabling informed patient care and heightened ...