Geographic AtrophyPhase 1NCT03777332
Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy
A Phase 1 study testing PEGCETACOPLAN (APL-2) in people with Geographic Atrophy, run by Apellis Pharmaceuticals, Inc. at 5 sites in United States.
Trial summary
Safety Assessment of APL-2 in Patients with Geographic Atrophy Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Geographic Atrophy
- What is being tested?
- PEGCETACOPLAN (APL-2)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 5 sitesUnited States
- Study dates
- 5 Nov 2018to 1 Apr 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The study eye must meet all inclusion criteria. If both eyes meet the inclusion criteria, the eye with the worst normal luminance best-corrected visual acuity (NL-BCVA) at the screening visit will be designated as the study eye. If both eyes have the same visual acuity, the eye with the larger GA lesion will be selected as the study eye. If both eyes have the same GA lesion size, the right eye will be selected as the study eye.
- Ocular- specific inclusion criteria apply to the study eye only, unless otherwise specified.
- Age ≥ 60 years.
- Normal Luminance best corrected visual acuity of between 35 and 5 letters using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately between 20/200 and 20/800 Snellen equivalent).
- Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center.
- The GA lesion must meet the following criteria as determined by the central reading center's assessment of Fundus Autofluorescence (FAF) imaging at screening:
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- Total GA area must be ≥ 2.5 (1 disk areas [DA]).
- If GA is multifocal, at least one focal lesion must be ≥ 1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 4a.
- Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the Investigator.
- Female subjects must be:
- Women of non-child-bearing potential (WONCBP), or
- Women of child-bearing 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.
- Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study.
- Willing and able to give informed consent and to comply with the study procedures and assessments.
- Ocular-specific inclusion criteria apply to the study eye only.
- Participated in the APL2-303 (Derby, NCT03525613) or APL2-304 (Oaks, NCT03525600) study with completion of the Month 24 visit and have been invited to participate in this study.
- Subjects who did not discontinue treatment but missed the Month 24 visit are also eligible to participate in this study.
- Clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the investigator.
- Female subjects must be:
- Women of non-child-bearing potential (WONCBP), or
- Women of child-bearing 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 90 days after their last dose of pegcetacoplan, and refrain from breastfeeding for the duration of the study.
- Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and for 90 days after their last dose of pegcetacoplan.
- Willing and able to give informed consent to comply with the study procedures and assessments
Who cannot join
- Ocular specific exclusion criteria apply to the study eye only, unless otherwise specified.
- GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye.
- Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm.
- Any history or active choroidal neovascularization (CNV), associated with AMD or any other cause, including any evidence of retinal pigment epithelium rips or evidence of neovascularization anywhere based on SD-OCT imaging and/or fluorescein angiography as assessed by the Reading Center.
- 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 (e.g. clinically significant epiretinal membrane [ERM], 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.
- Intraocular surgery (including lens replacement surgery) within 3 months prior to enrollment.
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- History of laser therapy in the macular region.
- History of intraocular injection within 3 months prior to the screening visit.
- Aphakia or absence of the posterior capsule. Note: Yttrium-Aluminum Garnet YAG laser posterior capsulotomy for posterior capsule opacification done at least 60 days prior to screening is not exclusionary.
- 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.
- Any contraindication to IVT injection including current ocular or periocular infection.
- Participation in any systemic experimental treatment or any other systemic investigational new drug including within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of study treatment. Note: clinical trials solely involving observation, over-the-counter vitamins, supplements, or diets are not exclusionary.
- Medical or psychiatric conditions that, in the opinion of the Investigator, make consistent follow-up over the 60-month treatment period unlikely, or would make the subject an unsafe study candidate.
- Any screening laboratory value (hematology, serum chemistry or urinalysis) that in the opinion of the Investigator is clinically significant and not suitable for study participation.
- Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to pegcetacoplan or any of the excipients in pegcetacoplan solution.
- Ocular-specific exclusion criteria apply to the study eye only, unless otherwise specified.
- Subjects who discontinued the study drug prior to Month 24 and remained in the APL2- 303 (Derby, NCT 03525613) or APL2-304 (Oaks, NCT03525600 study for safety assessments. Temporary pause of the study drug is not exclusionary.
- Presence of an active ocular disease that, in the opinion of the investigator, compromises or confounds visual function, including, but not limited to, macular hole or other macular diseases (eg, clinically significant epiretinal membrane). Benign conditions in the opinion of the investigator such as peripheral retinal dystrophy are not exclusionary.
- Any contraindication to IVT injection including current ocular or periocular infection.
- Medical or psychiatric conditions that, in the opinion of the investigator, is clinically significant and not suitable for study participation or consistent follow-up.
- Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to pegcetacoplan or any of the excipients in pegcetacoplan solution.
- Pregnancy, breastfeeding, or positive pregnancy test.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
California Retina Consultants
Bakersfield, California, 93309
Retina Vitreous Associates Medical Group
Beverly Hills, California, 90211
Ophthalmic Consultants of Boston
Boston, Massachusetts, 02114
The Retina Partners
Encino, California, 91436
Retina Consultants of Houston
The Woodlands, Texas, 77384
About this study
Official title: A 24-Month Phase Ib Multi-Center, Open Label, Single Arm Study to Evaluate the Safety of Intravitreal APL-2 Therapy in Patients Diagnosed With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Safety Assessment of APL-2 in Patients with Geographic Atrophy
- Conditions
- Geographic Atrophy
- Being tested
- PEGCETACOPLAN (APL-2)
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PEGCETACOPLAN (APL-2)Drug
Complement (C3) Inhibitor
| Study group | Role | What they receive |
|---|---|---|
| (Cohort 1) Pegcetacoplan, 15 mg/100 μL, monthly for up to 60 months | Experimental | Not described Receives: Drug: PEGCETACOPLAN (APL-2) |
| (Cohort 2) Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months | Experimental | Not described Receives: Drug: PEGCETACOPLAN (APL-2) |
| (Cohort 2) Pegcetacoplan, 15 mg/100 μL, every other month for up to 36 months | Experimental | Not described Receives: Drug: PEGCETACOPLAN (APL-2) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs)
Measured: 25 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2018
- Study start5 Nov 2018
- Primary completion1 Apr 2021
- Study completion1 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03777332
- Sponsor's protocol ID
- APL2-103
- Status last verified
- Nov 2021
- First submitted
- 14 Dec 2018
- Last updated
- 10 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Wykoff CC, Holz FG, Chiang A, Boyer D, Dhoot DS, Loewenstein A, Mones J, Heier J, Abbey AM, Singerman LJ, Vajzovic L, Lin J, Li C, Vilupuru A, Baumal CR; OAKS, DERBY, and GALE Investigators. Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE. Am J Ophthalmol. 2025 Aug;276:350-364. doi: 10.1016/j.ajo.2025.04.016. Epub 2025 Apr 23.
PubMed 40280279
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 24-Month Phase Ib Multi-Center, Open Label, Single Arm Study to Evaluate the Safety of Intravitreal APL-2 Therapy in Patients Diagnosed With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
- ClinicalTrials.gov ID
- NCT03777332
- Acronym / sponsor ID
- APL2-103
- Conditions
- Geographic Atrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Apellis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Nov 2018 (actual)
- Primary completion
- 1 Apr 2021 (actual)
- Study completion
- 1 Apr 2021 (actual)
- First posted
- 17 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Safety Assessment of APL-2 in Patients with Geographic Atrophy
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| (Cohort 1) Pegcetacoplan, 15 mg/100 μL, monthly for up to 60 months | Experimental | Drug: PEGCETACOPLAN (APL-2) |
| (Cohort 2) Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months | Experimental | Drug: PEGCETACOPLAN (APL-2) |
| (Cohort 2) Pegcetacoplan, 15 mg/100 μL, every other month for up to 36 months | Experimental | Drug: PEGCETACOPLAN (APL-2) |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- The study eye must meet all inclusion criteria. If both eyes meet the inclusion criteria, the eye with the worst normal luminance best-corrected visual acuity (NL-BCVA) at the screening visit will be designated as the study eye. If both eyes have the same visual acuity, the eye with the larger GA lesion will be selected as the study eye. If both eyes have the same GA lesion size, the right eye will be selected as the study eye.
- Ocular- specific inclusion criteria apply to the study eye only, unless otherwise specified.
- Age ≥ 60 years.
- Normal Luminance best corrected visual acuity of between 35 and 5 letters using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately between 20/200 and 20/800 Snellen equivalent).
- Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center.
Key exclusion criteria (22 listed)
- Ocular specific exclusion criteria apply to the study eye only, unless otherwise specified.
- GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye.
- Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm.
- Any history or active choroidal neovascularization (CNV), associated with AMD or any other cause, including any evidence of retinal pigment epithelium rips or evidence of neovascularization anywhere based on SD-OCT imaging and/or fluorescein angiography as assessed by the Reading Center.
- 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 (e.g. clinically significant epiretinal membrane [ERM], 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PEGCETACOPLAN (APL-2)Complement (C3) Inhibitor | Drug | (Cohort 1) Pegcetacoplan, 15 mg/100 μL, monthly for up to 60 months, (Cohort 2) Pegcetacoplan, 15 mg/100 μL, every other month for up to 36 months, (Cohort 2) Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months |
Primary outcome
Incidence and Severity of Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs)
Time frame: 25 Months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2021.
- Derived from this study: PubMed 40280279Wykoff CC, Holz FG, Chiang A, Boyer D, Dhoot DS, Loewenstein A, Mones J, Heier J, Abbey AM, Singerman LJ, Vajzovic L, Lin J, Li C, Vilupuru A, Baumal CR; OAKS, DERBY, and GALE Investigators. Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE. Am J Ophthalmol. 2025 Aug;276:350-364. doi: 10.1016/j.ajo.2025.04.016. Epub 2025 Apr 23.
Related trials for Geographic Atrophy
Active, not recruitingPhase 1/2
Phase 1/2 Study of VOY-101 in Patients With Advanced Non-Neovascular Age-Related Macular Degeneration
Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Age-related Macular Degeneration (AMD), Geographic Atrophy (GA)
RecruitingPhase 1
Phase 1 Study of C.001 in Retinal Degeneration
Geographic Atrophy, Stargardt Disease
RecruitingPhase 2/3
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Geographic Atrophy, Macular Degeneration
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PEGCETACOPLAN (APL-2) in people with Geographic Atrophy, run by Apellis Pharmaceuticals, Inc. at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 23 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs) (time frame: 25 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03777332, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Nov 2021.