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CompletedNCT04770545GALEUpdated Oct 28, 2026Results posted

An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD

A Phase 3 interventional study of PEGCETACOPLAN (APL-2) in Geographic Atrophy Secondary to Age-related Macular Degeneration, sponsored by Apellis Pharmaceuticals, Inc.. Completed at 175 sites in 15 countries. Open to participants aged 60 Years and older. Per ClinicalTrials.gov, last updated 2026-10-28.

Sponsored by Apellis Pharmaceuticals, Inc. (part of Biogen) · Phase 3, Interventional, and Treatment

Updated Oct 28, 2026Results postedStudy documents posted+5 moreGo to Updates ↓
Phase
Phase 3
Study type
Interventional
Enrollment
792
Allocation
Non-randomized
Ages
60 Years and older
Sex
All
01

Study summary

This is a phase 3, open-label, multicenter, extension study to evaluate the long-term safety and efficacy of pegcetacoplan (APL-2) in subjects with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) who participated in Study APL2-103 (NCT03777332) or completed the treatment at Month 24 of either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600).

02

Conditions studied

  • Geographic Atrophy Secondary to Age-related Macular Degeneration
03

In context

Geographic Atrophy

188 studies on the registry are indexed under Geographic Atrophy; 52 are open to participants now.

This study's enrollment of 792 is above the median of 70 across 141 interventional studies indexed under Geographic Atrophy.

Browse Geographic Atrophy studies →

Lead sponsor

Apellis Pharmaceuticals, Inc. is the lead sponsor of 28 studies on the registry; 3 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.

First submitted before Apellis Pharmaceuticals, Inc. became part of Biogen.

Browse Apellis Pharmaceuticals, Inc. studies →All Biogen studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Oct 28, 2026. See what changed

Ages eligible
60 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

Participants are eligible to be included in the study only if all of the following criteria apply:

Ocular-specific inclusion criteria apply to the study eye only.

  • Subjects must have participated in APL2-103 (NCT03777332) or completed the treatment at Month 24 of either APL2-303 (Derby, NCT03525613) or APL2-304 (Oaks, NCT03525600).

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

    • Subjects who did not fully stop study drug treatment, but missed the visit at Month 24, can also participate in the study. However, to participate, subjects must be seen within 60 days from the last day of the period allowed for the 24 months visit in the previous study.
  • The eyes of subjects must have transparency to permit visualization of parts inside the eye such as the retina and subjects must be able to look steadily at a provided target allowing the doctor to get good quality pictures from the eye.

  • Female subjects must be:

    • Women that cannot have children, or
    • 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.
  • Males with female partners who can get pregnant must also agree to use ways to avoid pregnancy and agree not to donate sperm while in the study or until 90 days after administering the last dose of the study medication.
  • Agree to participate in the study by signing the consent document providing information about the study; and take part in all tests and assessments as required.

Exclusion criteria

Exclusion Criteria:

Subjects cannot take part in the study if the eye that will be treated during the study currently meets any of the following conditions:

  • Participants in APL2 303/304 studies who fully stopped study drug treatment before the visit in month 24 but remain in the study to observe the study medication's safety. Those subjects who temporarily paused study drug treatment are still eligible to participate.
  • If, according to your doctor you currently have any disease affecting your eyes that could interfere with your vision, including diseases that affect your retina or macula (the center of the back of your eyes). And, if according to your doctor, the disease in your eyes is benign and does not interfere with the study (e.g. diseases that affects the periphery of the retina), you are also eligible to take part in the study.
  • If, according to your doctor you currently have any inflammation/infection in or around your eyes that could prohibit you from receiving an injection inside your eyes.
  • 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 have known allergies to fluorescein sodium, a solution that is injected into your body for eye testing; or to pegcetacoplan (the study drug) or any inactive substances in pegcetacoplan solution.
  • If you currently are pregnant, breastfeed or have a positive pregnancy test.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
792 participants (actual)

Study arms

  • Experimental
    Monthly: Pegcetacoplan/ Pegcetacoplan

    Participants who received monthly intravitreal (IVT) pegcetacoplan (15 milligrams \[mg\]/100 microlitres \[μL\]) in the antecedent studies (APL2-103 \[NCT03777332\], APL2-303 \[NCT03525600\], and APL2-304 \[NCT03525613\]) continued to receive monthly IVT pegcetacoplan (15 mg/100 μL) for up to 36 months.

    Drug: PEGCETACOPLAN (APL-2)

  • Experimental
    EOM: Pegcetacoplan/Pegcetacoplan

    Participants who received IVT pegcetacoplan (15 mg/100 μL) every other month (EOM) in the antecedent studies (APL2-103 \[NCT03777332\], APL2-303 \[NCT03525600\], and APL2-304 \[NCT03525613\]) continued to receive IVT pegcetacoplan (15 mg/100 μL) EOM for up to 36 months.

    Drug: PEGCETACOPLAN (APL-2)

  • Experimental
    Monthly: Sham/Pegcetacoplan

    Participants who received sham monthly in the antecedent studies (APL2-103 \[NCT03777332\], APL2-303 \[NCT03525600\], and APL2-304 \[NCT03525613\]) received monthly IVT pegcetacoplan (15 mg/100 μL) for up to 36 months.

    Drug: PEGCETACOPLAN (APL-2)

  • Experimental
    EOM: Sham/Pegcetacoplan

    Participants who received sham EOM in the antecedent studies (APL2-103 \[NCT03777332\], APL2-303 \[NCT03525600\], and APL2-304 \[NCT03525613\]) received IVT pegcetacoplan (15 mg/100 μL) EOM for up to 36 months.

    Drug: PEGCETACOPLAN (APL-2)

Interventions

06

What researchers measure

Primary outcomes

  1. Number of Participants Who Experienced at Least One Ocular and Systemic Adverse Events (AEs)

    An AE is any untoward medical occurrence associated with use of a drug in humans, whether or not it is considered study drug-related. An AE can, therefore, be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of an investigational product, whether considered related to the investigational product.

    Time frame: From Day 1 up to Month 36

Secondary outcomes

  1. Change From Baseline in Total Area of Geographic Atrophy (GA) Lesion(s) in the Study Eye

    The change in GA lesion area was measured by a qualified central reading center based on fundus autofluorescence (FAF) images.

    Time frame: Baseline, Months 12, 24 and 36

  2. Change From Baseline in the Rate of GA Lesion Growth in the Study Eye

    The mean rate of change in GA area was analyzed by use of a piecewise linear mixed effect model assuming time as continuous and piecewise linear in FAS based on FAF images.

    Time frame: Baseline, Months 12, 24 and 36

  3. Change From Baseline in Normal-Luminance Best-Corrected Visual Acuity (NL-BCVA) Score of the Study Eye

    The NL-BCVA was assessed by early treatment diabetic retinopathy study (ETDRS) chart prior to dilating the eyes at a starting distance of 4 meters and ranged from 0 (least score) to 100 (best score). If the 4-meter score was \>19 letters read correctly, the visual acuity score was the sum of total letters correctly read at 4 meters plus the addition of 30. If the 4-meter score was ≤19 letters read correctly, the visual acuity score was the sum of total letters read correctly at 4 meters and total letters read correctly at the 1-meter distance. If no letters were read correctly at either the 4-meter distance or the 1-meter distance, the visual acuity score was 0. A negative change from baseline indicates loss in visual acuity.

    Time frame: Baseline, Months 12, 24 and 36

  4. Change From Baseline in Low-Luminance Best-Corrected Visual Acuity (LL-BCVA) Score of the Study Eye

    The LL-BCVA was assessed by early treatment diabetic retinopathy study (ETDRS) chart prior to dilating the eyes at a starting distance of 4 meters and ranged from 0 (least score) to 100 (best score). It was measured by placing a neutral density trial lens causing a reduction of 2.0 log units in luminance. If the 4-meter score was \>19 letters read correctly, the visual acuity score was the sum of total letters correctly read at 4 meters plus the addition of 30. If the 4-meter score was ≤19 letters read correctly, the visual acuity score was the sum of total letters read correctly at 4 meters and total letters read correctly at the 1-meter distance. If no letters were read correctly at either the 4-meter distance or the 1-meter distance, the visual acuity score was 0. A negative change from baseline indicates loss in visual acuity.

    Time frame: Baseline, Months 12, 24 and 36

  5. Change From Baseline in Monocular Maximum Corrected Reading Speed of the Study Eye

    The maximum reading speed of the study eye was calculated per Minnesota Low-Vision Reading Test (MNRead) or Radner user manuals, with adjustment for reading inaccuracy. An additional step to cap resulting reading speed values at a maximum of 300 words per minute was implemented. Maximum reading speed was calculated as the mean of the three highest nonzero reading speeds (or two, or one value, as available), except when all words per minute were calculated as 0: then the maximum reading speed was calculated as 0. A negative change from baseline indicated a decrease in the monocular reading speed; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  6. Change From Baseline in Binocular Maximum Corrected Reading Speed

    The maximum reading speed was calculated per MNRead or Radner user manuals, with adjustment for reading inaccuracy. An additional step to cap resulting reading speed values at a maximum of 300 words per minute was implemented. Maximum reading speed was calculated as the mean of the three highest nonzero reading speeds (or two, or one value, as available), except when all words per minute were calculated as 0: then the maximum reading speed was calculated as 0. A negative change from baseline indicated a decrease in the binocular reading speed; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  7. Change From Baseline in Number of Scotomatous Points of the Study Eye

    The number of absolute scotomatous points was determined from mesopic microperimetry as an assessment of the macular functional response.

    Time frame: Baseline, Months 12, 24 and 36

  8. Change From Baseline in Macular Sensitivity of the Study Eye

    Macular sensitivity was assessed by mesopic microperimetry. A negative change from baseline indicates a decrease in the macular sensitivity.

    Time frame: Baseline, Months 12, 24 and 36

  9. Change From Baseline in 95% Bivariate Contour Ellipse Area (BCEA) Degrees Squared (Deg²) of the Study Eye

    Microperimetry was conducted to assess the bivariate contour ellipse area 95%.

    Time frame: Baseline, Months 12, 24 and 36

  10. Mean Change From Baseline in Functional Reading Independence (FRI) Index Score

    The FRI index is an individualized assessment of functional reading independence. The questionnaire has 7 items with one total index score. Scores derived from the index range from 1 (unable to do independently) to 4 (total independence) with higher scores indicating higher functional reading independence. A negative change from baseline indicated a decrease in the FRI; disease worsening. Least squares mean was calculated using a mixed effect model for repeated measure (MMRM) model.

    Time frame: Baseline, Months 12, 24 and 36

  11. Change From Baseline in National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) Composite Score

    The NEI-VFQ-25 is an interviewer-administered questionnaire designed to assess participant-reported visual function. It is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being (general health, general vision, ocular pain, near activities, distance activities, social functioning, mental health, role difficulties, dependency, driving, color vision, and peripheral vision). Scoring yields 12 subscales based on the 12 domains covered in the questionnaire. These scales are scored from 0 to 100 with higher scores indicating better visual function. A composite score is the average of the vision-targeted sub-scale scores, excluding the general health rating question. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  12. Change From Baseline in NEI VFQ-25 Near Activity Subscale Score

    The NEI-VFQ-25 is an interviewer-administered questionnaire designed to assess participant-reported visual function. It is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being (general health, general vision, ocular pain, near activities, distance activities, social functioning, mental health, role difficulties, dependency, driving, color vision, and peripheral vision). Scoring yields 12 subscales based on the 12 domains covered in the questionnaire. The possible score range is 0 to 100 with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening. Near activities are defined as reading ordinary print in newspapers, performing work or hobbies requiring near vision, or finding something on a crowded shelf.

    Time frame: Baseline, Months 12, 24 and 36

  13. Change From APL2-GA-305 Baseline in NEI VFQ-25 Distance Activity Subscale Score

    The NEI-VFQ-25 is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being. A distance activity subscale score one of the domains is the average of 3 items: 8 (How much difficulty do you have reading street signs or the names of stores?), 9 (Because of your eyesight, how much difficulty do you have going down steps, stairs, or curbs in dim light or at night?) and 14 (Because of your eyesight, how much difficulty do you have going out to see movies, plays, or sports events?). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  14. Change From Baseline in NEI VFQ-25 Driving Subscale Score

    The NEI-VFQ-25 is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being. A driving subscale score one of the domains is the average of 3 items: 15c (If currently driving: How much difficulty do you have driving during the day time in familiar places?), 16 (How much difficulty do you have driving at night), and 16a (How much difficulty do you have driving in difficult conditions, such as in bad weather, during rush hour, on the freeway, or in city traffic?). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  15. Change From Baseline in National Eye Institute Visual Functioning Questionnaire 39-Item Version (NEI VFQ-39) Composite Score

    NEI-VFQ-25 includes 13 additional items under 7 subscales related to general health, general vision, near vision, distance vision, social function, driving, role limitations, well-being or distress and dependency that allow for the calculation of NEI-VFQ-39 scores. The NEI-VFQ composite score is obtained by averaging the vision-related subscales, excluding the general health rating question. The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  16. Change From Baseline in NEI VFQ-39 Near Activity Subscale Score

    NEI-VFQ-25 includes 13 additional items under 7 subscales, enabling calculation of NEI-VFQ-39 scores. NEI-VFQ near activity subscale score is obtained by averaging 6 items: 5 (How much difficulty do you have reading ordinary print in newspapers), 6 (How much difficulty do you have doing work/hobbies that require you to see up close?), 7 (Because of your eyesight, how much difficulty do you have finding something on a crowded shelf?), A3 (Wearing glasses, how much difficulty do you have reading the small print in a telephone book, on medicine bottle, or on legal forms?), A4 (Because of your eyesight, how much difficulty do you have figuring out bills you receive are accurate?) \& A5 (Because of your eyesight, how much difficulty do you have doing things like shaving, styling your hair or putting on makeup). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated reduced visual functioning; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

  17. Change From Baseline in NEI VFQ-39 Distance Activity Subscale Score

    NEI-VFQ-25 includes 13 additional items under 7 subscales, enabling calculation of NEI-VFQ-39 scores. NEI-VFQ near activity subscale score is obtained by averaging 6 items: 8 (How much difficulty do you have reading street signs/names of stores?), 9 (Because of your eyesight, how much difficulty do you have going down steps, stairs/ curbs in dim light/ at night?) and 14 (Because of your eyesight, how much difficulty do you have going out to see movies, plays/sports events?), A6 (Because of your eyesight, how much difficulty do you have recognizing people you know from across a room?), A7 (Because of your eyesight, how much difficulty do you have taking part in active sports/other outdoor activities?) \& A8 (Because of your eyesight, how much difficulty do you have seeing \& enjoying programs on TV?). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated reduced visual functioning; disease worsening.

    Time frame: Baseline, Months 12, 24 and 36

07

Results

Posted Oct 28, 2026

Participant flow

Participants took part in the study at multiple investigative sites from 04 March 2021 to 05 September 2025.

Participant flow — Overall Study
MilestoneMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Started250268129143
Completed1771798092
Not completed73894951
Withdrew: Consent withdrawal29452116
Withdrew: Death1519812
Withdrew: Adverse event101065
Withdrew: Reason not specified53510
Withdrew: Lost to follow-up7652
Withdrew: Physician decision7243
Withdrew: Non-compliance with study drug0302
Withdrew: Transitioned to commercially available drug0101

Outcome measures

PrimaryNumber of Participants Who Experienced at Least One Ocular and Systemic Adverse Events (AEs)

An AE is any untoward medical occurrence associated with use of a drug in humans, whether or not it is considered study drug-related. An AE can, therefore, be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of an investigational product, whether considered related to the investigational product.

Time frame:
From Day 1 up to Month 36
Reported as:
Count of participants · Participants
Number of Participants Who Experienced at Least One Ocular and Systemic Adverse Events (AEs)
ParticipantsMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Ocular AEs1611198377
Systemic AEs212213109110
SecondaryChange From Baseline in Total Area of Geographic Atrophy (GA) Lesion(s) in the Study Eye

The change in GA lesion area was measured by a qualified central reading center based on fundus autofluorescence (FAF) images.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · millimeter square (mm²)
Change From Baseline in Total Area of Geographic Atrophy (GA) Lesion(s) in the Study Eye
millimeter square (mm²)Monthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 121.29 (1.17 to 1.41)1.49 (1.36 to 1.63)1.37 (1.20 to 1.53)1.59 (1.40 to 1.77)
Change at Month 242.84 (2.66 to 3.03)2.91 (2.69 to 3.12)2.71 (2.49 to 2.93)3.00 (2.69 to 3.31)
Change at Month 364.38 (4.09 to 4.66)4.48 (4.16 to 4.79)3.91 (3.60 to 4.23)4.57 (4.19 to 4.96)
SecondaryChange From Baseline in the Rate of GA Lesion Growth in the Study Eye

The mean rate of change in GA area was analyzed by use of a piecewise linear mixed effect model assuming time as continuous and piecewise linear in FAS based on FAF images.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Mean · mm^2/year
Change From Baseline in the Rate of GA Lesion Growth in the Study Eye
mm^2/yearMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 121.32 (1.19 to 1.46)1.53 (1.39 to 1.66)1.36 (1.19 to 1.54)1.68 (1.47 to 1.88)
Change at Month 242.91 (2.70 to 3.13)2.97 (2.76 to 3.19)2.69 (2.43 to 2.95)3.20 (2.86 to 3.54)
Change at Month 364.46 (4.13 to 4.79)4.53 (4.21 to 4.86)3.86 (3.50 to 4.21)4.86 (4.41 to 5.31)
SecondaryChange From Baseline in Normal-Luminance Best-Corrected Visual Acuity (NL-BCVA) Score of the Study Eye

The NL-BCVA was assessed by early treatment diabetic retinopathy study (ETDRS) chart prior to dilating the eyes at a starting distance of 4 meters and ranged from 0 (least score) to 100 (best score). If the 4-meter score was \>19 letters read correctly, the visual acuity score was the sum of total letters correctly read at 4 meters plus the addition of 30. If the 4-meter score was ≤19 letters read correctly, the visual acuity score was the sum of total letters read correctly at 4 meters and total letters read correctly at the 1-meter distance. If no letters were read correctly at either the 4-meter distance or the 1-meter distance, the visual acuity score was 0. A negative change from baseline indicates loss in visual acuity.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · ETDRS letters score
Change From Baseline in Normal-Luminance Best-Corrected Visual Acuity (NL-BCVA) Score of the Study Eye
ETDRS letters scoreMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-4.4 (-5.8 to -2.9)-4.0 (-5.2 to -2.9)-4.4 (-6.2 to -2.6)-4.6 (-6.3 to -3.0)
Change at Month 24-8.0 (-9.7 to -6.2)-6.1 (-7.7 to -4.4)-8.0 (-10.4 to -5.7)-7.6 (-9.9 to -5.4)
Change at Month 36-12.5 (-14.6 to -10.5)-10.0 (-11.9 to -8.1)-13.0 (-16.0 to -10.1)-10.3 (-12.9 to -7.8)
SecondaryChange From Baseline in Low-Luminance Best-Corrected Visual Acuity (LL-BCVA) Score of the Study Eye

The LL-BCVA was assessed by early treatment diabetic retinopathy study (ETDRS) chart prior to dilating the eyes at a starting distance of 4 meters and ranged from 0 (least score) to 100 (best score). It was measured by placing a neutral density trial lens causing a reduction of 2.0 log units in luminance. If the 4-meter score was \>19 letters read correctly, the visual acuity score was the sum of total letters correctly read at 4 meters plus the addition of 30. If the 4-meter score was ≤19 letters read correctly, the visual acuity score was the sum of total letters read correctly at 4 meters and total letters read correctly at the 1-meter distance. If no letters were read correctly at either the 4-meter distance or the 1-meter distance, the visual acuity score was 0. A negative change from baseline indicates loss in visual acuity.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · ETDRS letters score
Change From Baseline in Low-Luminance Best-Corrected Visual Acuity (LL-BCVA) Score of the Study Eye
ETDRS letters scoreMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-3.0 (-4.0 to -2.1)-2.0 (-3.1 to -1.0)-3.1 (-4.5 to -1.7)-4.4 (-5.9 to -2.8)
Change at Month 24-5.1 (-6.4 to -3.7)-2.9 (-4.3 to -1.5)-3.8 (-5.7 to -1.8)-5.9 (-7.7 to -4.1)
Change at Month 36-6.7 (-8.2 to -5.2)-5.8 (-7.4 to -4.3)-5.9 (-8.0 to -3.9)-7.4 (-9.5 to -5.4)
SecondaryChange From Baseline in Monocular Maximum Corrected Reading Speed of the Study Eye

The maximum reading speed of the study eye was calculated per Minnesota Low-Vision Reading Test (MNRead) or Radner user manuals, with adjustment for reading inaccuracy. An additional step to cap resulting reading speed values at a maximum of 300 words per minute was implemented. Maximum reading speed was calculated as the mean of the three highest nonzero reading speeds (or two, or one value, as available), except when all words per minute were calculated as 0: then the maximum reading speed was calculated as 0. A negative change from baseline indicated a decrease in the monocular reading speed; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · words per minute
Change From Baseline in Monocular Maximum Corrected Reading Speed of the Study Eye
words per minuteMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-8.2 (-13.4 to -3.0)-9.4 (-14.1 to -4.8)-9.6 (-15.4 to -3.8)-8.7 (-12.4 to -5.1)
Change at Month 24-16.4 (-21.1 to -11.8)-16.3 (-20.2 to -12.3)-17.8 (-24.0 to -11.5)-13.5 (-18.2 to -8.9)
Change at Month 36-22.5 (-26.7 to -18.3)-19.6 (-25.0 to -14.3)-25.9 (-32.6 to -19.1)-24.8 (-30.3 to -19.2)
SecondaryChange From Baseline in Binocular Maximum Corrected Reading Speed

The maximum reading speed was calculated per MNRead or Radner user manuals, with adjustment for reading inaccuracy. An additional step to cap resulting reading speed values at a maximum of 300 words per minute was implemented. Maximum reading speed was calculated as the mean of the three highest nonzero reading speeds (or two, or one value, as available), except when all words per minute were calculated as 0: then the maximum reading speed was calculated as 0. A negative change from baseline indicated a decrease in the binocular reading speed; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · words per minute
Change From Baseline in Binocular Maximum Corrected Reading Speed
words per minuteMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-11.6 (-16.6 to -6.6)-14.5 (-19.0 to -9.9)-12.9 (-19.1 to -6.6)-11.4 (-16.9 to -5.9)
Change at Month 24-22.5 (-27.2 to -17.7)-23.4 (-28.3 to -18.5)-21.0 (-28.7 to -13.4)-21.5 (-27.9 to -15.1)
Change at Month 36-33.2 (-37.9 to -28.4)-30.9 (-37.6 to -24.3)-32.9 (-41.0 to -24.9)-36.8 (-44.0 to -29.5)
SecondaryChange From Baseline in Number of Scotomatous Points of the Study Eye

The number of absolute scotomatous points was determined from mesopic microperimetry as an assessment of the macular functional response.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · number of scotomatous points
Change From Baseline in Number of Scotomatous Points of the Study Eye
number of scotomatous pointsMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 123.3 (2.0 to 4.6)4.5 (3.3 to 5.7)4.3 (2.6 to 6.1)5.8 (4.4 to 7.3)
Change at Month 246.5 (5.0 to 8.1)8.4 (6.9 to 9.8)7.1 (5.0 to 9.1)9.1 (7.3 to 10.9)
Change at Month 369.8 (7.9 to 11.7)11.9 (10.2 to 13.6)11.0 (8.5 to 13.6)12.4 (10.2 to 14.7)
SecondaryChange From Baseline in Macular Sensitivity of the Study Eye

Macular sensitivity was assessed by mesopic microperimetry. A negative change from baseline indicates a decrease in the macular sensitivity.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · decibel
Change From Baseline in Macular Sensitivity of the Study Eye
decibelMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-0.97 (-1.33 to -0.61)-1.37 (-1.69 to -1.05)-1.74 (-2.16 to -1.32)-1.81 (-2.25 to -1.37)
Change at Month 24-2.06 (-2.48 to -1.64)-2.47 (-2.93 to -2.00)-2.04 (-2.56 to -1.52)-2.57 (-3.04 to -2.11)
Change at Month 36-3.18 (-3.62 to -2.74)-3.43 (-3.92 to -2.94)-3.68 (-4.38 to -2.98)-3.86 (-4.48 to -3.24)
SecondaryChange From Baseline in 95% Bivariate Contour Ellipse Area (BCEA) Degrees Squared (Deg²) of the Study Eye

Microperimetry was conducted to assess the bivariate contour ellipse area 95%.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · deg²
Change From Baseline in 95% Bivariate Contour Ellipse Area (BCEA) Degrees Squared (Deg²) of the Study Eye
deg²Monthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 126.40 (-1.46 to 14.26)9.02 (2.49 to 15.56)3.02 (-4.21 to 10.25)4.47 (-2.74 to 11.67)
Change at Month 245.17 (-1.81 to 12.15)15.05 (6.85 to 23.25)0.55 (-6.17 to 7.26)9.95 (0.87 to 19.03)
Change at Month 3612.78 (5.84 to 19.73)8.90 (2.48 to 15.31)7.64 (0.18 to 15.10)9.52 (-1.73 to 20.77)
SecondaryMean Change From Baseline in Functional Reading Independence (FRI) Index Score

The FRI index is an individualized assessment of functional reading independence. The questionnaire has 7 items with one total index score. Scores derived from the index range from 1 (unable to do independently) to 4 (total independence) with higher scores indicating higher functional reading independence. A negative change from baseline indicated a decrease in the FRI; disease worsening. Least squares mean was calculated using a mixed effect model for repeated measure (MMRM) model.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · score on a scale
Mean Change From Baseline in Functional Reading Independence (FRI) Index Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-0.139 (-0.203 to -0.074)-0.198 (-0.267 to -0.130)-0.040 (-0.128 to 0.048)-0.155 (-0.245 to -0.064)
Change at Month 24-0.245 (-0.327 to -0.163)-0.330 (-0.409 to -0.252)-0.147 (-0.256 to -0.039)-0.246 (-0.365 to -0.127)
Change at Month 36-0.439 (-0.559 to -0.319)-0.403 (-0.514 to -0.293)-0.331 (-0.474 to -0.189)-0.475 (-0.626 to -0.324)
SecondaryChange From Baseline in National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) Composite Score

The NEI-VFQ-25 is an interviewer-administered questionnaire designed to assess participant-reported visual function. It is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being (general health, general vision, ocular pain, near activities, distance activities, social functioning, mental health, role difficulties, dependency, driving, color vision, and peripheral vision). Scoring yields 12 subscales based on the 12 domains covered in the questionnaire. These scales are scored from 0 to 100 with higher scores indicating better visual function. A composite score is the average of the vision-targeted sub-scale scores, excluding the general health rating question. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · score on a scale
Change From Baseline in National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) Composite Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-2.3 (-3.5 to -1.1)-3.4 (-4.7 to -2.1)-2.1 (-3.7 to -0.4)-2.1 (-3.7 to -0.5)
Change at Month 24-5.8 (-7.4 to -4.3)-6.0 (-7.6 to -4.5)-4.3 (-6.4 to -2.2)-4.4 (-6.6 to -2.2)
Change at Month 36-7.4 (-9.3 to -5.5)-9.5 (-11.5 to -7.6)-6.7 (-9.4 to -3.9)-10.3 (-12.8 to -7.8)
SecondaryChange From Baseline in NEI VFQ-25 Near Activity Subscale Score

The NEI-VFQ-25 is an interviewer-administered questionnaire designed to assess participant-reported visual function. It is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being (general health, general vision, ocular pain, near activities, distance activities, social functioning, mental health, role difficulties, dependency, driving, color vision, and peripheral vision). Scoring yields 12 subscales based on the 12 domains covered in the questionnaire. The possible score range is 0 to 100 with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening. Near activities are defined as reading ordinary print in newspapers, performing work or hobbies requiring near vision, or finding something on a crowded shelf.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · score on a scale
Change From Baseline in NEI VFQ-25 Near Activity Subscale Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-4.8 (-6.9 to -2.7)-5.3 (-7.3 to -3.3)-3.1 (-6.0 to -0.3)-3.3 (-6.1 to -0.4)
Change at Month 24-8.0 (-10.4 to -5.7)-6.9 (-9.1 to -4.7)-6.4 (-9.4 to -3.4)-3.9 (-7.2 to -0.6)
Change at Month 36-7.8 (-10.9 to -4.7)-11.6 (-14.9 to -8.4)-8.5 (-12.9 to -4.1)-10.3 (-14.2 to -6.3)
SecondaryChange From APL2-GA-305 Baseline in NEI VFQ-25 Distance Activity Subscale Score

The NEI-VFQ-25 is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being. A distance activity subscale score one of the domains is the average of 3 items: 8 (How much difficulty do you have reading street signs or the names of stores?), 9 (Because of your eyesight, how much difficulty do you have going down steps, stairs, or curbs in dim light or at night?) and 14 (Because of your eyesight, how much difficulty do you have going out to see movies, plays, or sports events?). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · score on a scale
Change From APL2-GA-305 Baseline in NEI VFQ-25 Distance Activity Subscale Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-2.9 (-5.1 to -0.7)-3.7 (-5.9 to -1.6)-1.9 (-4.5 to 0.7)-5.0 (-8.2 to -1.8)
Change at Month 24-8.0 (-10.8 to -5.3)-6.1 (-8.7 to -3.4)-4.5 (-8.2 to -0.9)-9.1 (-12.9 to -5.4)
Change at Month 36-10.9 (-14.1 to -7.7)-11.9 (-15.6 to -8.2)-6.7 (-11.7 to -1.7)-15.3 (-19.5 to -11.1)
SecondaryChange From Baseline in NEI VFQ-25 Driving Subscale Score

The NEI-VFQ-25 is a 25-item questionnaire with a composite score and covers 12 domains of functional health status and well-being. A driving subscale score one of the domains is the average of 3 items: 15c (If currently driving: How much difficulty do you have driving during the day time in familiar places?), 16 (How much difficulty do you have driving at night), and 16a (How much difficulty do you have driving in difficult conditions, such as in bad weather, during rush hour, on the freeway, or in city traffic?). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Least squares mean · score on a scale
Change From Baseline in NEI VFQ-25 Driving Subscale Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-5.6 (-7.8 to -3.3)-7.6 (-9.7 to -5.4)-5.9 (-10.0 to -1.8)-4.7 (-7.8 to -1.6)
Change at Month 24-12.9 (-15.6 to -10.3)-12.4 (-15.2 to -9.6)-10.2 (-15.2 to -5.1)-10.3 (-14.6 to -6.0)
Change at Month 36-16.5 (-19.9 to -13.0)-17.2 (-20.6 to -13.9)-15.3 (-21.4 to -9.3)-17.0 (-22.6 to -11.5)
SecondaryChange From Baseline in National Eye Institute Visual Functioning Questionnaire 39-Item Version (NEI VFQ-39) Composite Score

NEI-VFQ-25 includes 13 additional items under 7 subscales related to general health, general vision, near vision, distance vision, social function, driving, role limitations, well-being or distress and dependency that allow for the calculation of NEI-VFQ-39 scores. The NEI-VFQ composite score is obtained by averaging the vision-related subscales, excluding the general health rating question. The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated a decrease in visual functioning; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Mean · score on a scale
Change From Baseline in National Eye Institute Visual Functioning Questionnaire 39-Item Version (NEI VFQ-39) Composite Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-2.0 ± 8.91-3.2 ± 10.20-2.8 ± 8.65-1.7 ± 9.57
Change at Month 24-5.7 ± 12.44-5.7 ± 10.67-4.7 ± 10.74-4.3 ± 12.75
Change at Month 36-7.3 ± 13.34-9.6 ± 11.93-7.8 ± 11.82-11.6 ± 12.42
SecondaryChange From Baseline in NEI VFQ-39 Near Activity Subscale Score

NEI-VFQ-25 includes 13 additional items under 7 subscales, enabling calculation of NEI-VFQ-39 scores. NEI-VFQ near activity subscale score is obtained by averaging 6 items: 5 (How much difficulty do you have reading ordinary print in newspapers), 6 (How much difficulty do you have doing work/hobbies that require you to see up close?), 7 (Because of your eyesight, how much difficulty do you have finding something on a crowded shelf?), A3 (Wearing glasses, how much difficulty do you have reading the small print in a telephone book, on medicine bottle, or on legal forms?), A4 (Because of your eyesight, how much difficulty do you have figuring out bills you receive are accurate?) \& A5 (Because of your eyesight, how much difficulty do you have doing things like shaving, styling your hair or putting on makeup). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated reduced visual functioning; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Mean · score on a scale
Change From Baseline in NEI VFQ-39 Near Activity Subscale Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-3.7 ± 14.55-4.6 ± 13.79-3.5 ± 14.16-4.1 ± 14.67
Change at Month 24-8.1 ± 17.87-6.3 ± 15.25-6.7 ± 15.05-6.3 ± 16.70
Change at Month 36-8.0 ± 19.94-11.7 ± 18.62-9.1 ± 21.07-13.3 ± 16.43
SecondaryChange From Baseline in NEI VFQ-39 Distance Activity Subscale Score

NEI-VFQ-25 includes 13 additional items under 7 subscales, enabling calculation of NEI-VFQ-39 scores. NEI-VFQ near activity subscale score is obtained by averaging 6 items: 8 (How much difficulty do you have reading street signs/names of stores?), 9 (Because of your eyesight, how much difficulty do you have going down steps, stairs/ curbs in dim light/ at night?) and 14 (Because of your eyesight, how much difficulty do you have going out to see movies, plays/sports events?), A6 (Because of your eyesight, how much difficulty do you have recognizing people you know from across a room?), A7 (Because of your eyesight, how much difficulty do you have taking part in active sports/other outdoor activities?) \& A8 (Because of your eyesight, how much difficulty do you have seeing \& enjoying programs on TV?). The possible score range is 0 to 100, with higher scores indicating better visual function. A negative change from baseline indicated reduced visual functioning; disease worsening.

Time frame:
Baseline, Months 12, 24 and 36
Reported as:
Mean · score on a scale
Change From Baseline in NEI VFQ-39 Distance Activity Subscale Score
score on a scaleMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
Change at Month 12-2.9 ± 13.98-3.6 ± 15.38-1.6 ± 11.94-4.7 ± 15.78
Change at Month 24-9.0 ± 20.22-5.1 ± 16.31-4.8 ± 14.32-10.0 ± 18.95
Change at Month 36-11.9 ± 20.82-10.6 ± 16.70-8.1 ± 16.15-17.0 ± 19.77

Adverse events

Collected over From Day 1 up to Month 36. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Monthly: Pegcetacoplan/Pegcetacoplan14/250 (5.6%)105/250 (42%)193/250 (77.2%)
EOM: Pegcetacoplan/Pegcetacoplan19/268 (7.1%)103/268 (38.4%)176/268 (65.7%)
Monthly: Sham/Pegcetacoplan7/129 (5.4%)50/129 (38.8%)102/129 (79.1%)
EOM: Sham/Pegcetacoplan12/143 (8.4%)54/143 (37.8%)102/143 (71.3%)
Most frequent serious events
Showing 10 of 304
Most frequent serious events
EventMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
PneumoniaInfections and infestations9/25010/2685/1294/143
Atrial fibrillationCardiac disorders9/2506/2684/1294/143
COVID-19Infections and infestations9/2502/2684/1291/143
Acute respiratory failureRespiratory, thoracic and mediastinal disorders2/2502/2683/1295/143
SepsisInfections and infestations2/2505/2684/1291/143
AnaemiaBlood and lymphatic system disorders6/2500/2681/1291/143
FallInjury, poisoning and procedural complications6/2503/2683/1291/143
Cardiac failure congestiveCardiac disorders4/2503/2683/1293/143
Urinary tract infectionInfections and infestations3/2503/2683/1292/143
Acute kidney injuryRenal and urinary disorders2/2503/2683/1292/143
Most frequent other events
Showing 10 of 28
Most frequent other events
EventMonthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/Pegcetacoplan
COVID-19Infections and infestations54/25054/26833/12930/143
FallInjury, poisoning and procedural complications29/25022/26821/12912/143
Neovascular age-related macular degenerationEye disorders39/25018/26811/12912/143
Urinary tract infectionInfections and infestations28/25032/26818/12915/143
Conjunctival haemorrhageEye disorders22/25015/26817/12915/143
Vitreous floatersEye disorders20/25012/26817/12911/143
Retinal haemorrhageEye disorders29/25020/26815/12910/143
Intraocular pressure increasedInvestigations28/25024/26814/1295/143
Posterior capsule opacificationEye disorders16/25011/26814/1299/143
HypertensionVascular disorders26/25019/26814/1298/143

Baseline characteristics

The safety set consisted of all enrolled participants who received at least one injection of Pegcetacoplan in APL2-GA-305 (NCT04770545).

Age, Continuous
Age, Continuous(years)Monthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/PegcetacoplanTotal
Mean79.5 ± 6.8079.6 ± 7.2679.6 ± 7.5279.5 ± 7.0679.6 ± 7.11
Sex: Female, Male
Sex: Female, Male(Participants)Monthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/PegcetacoplanTotal
Female1471597789472
Male1031095254318
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Monthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/PegcetacoplanTotal
Hispanic or Latino2324111472
Not Hispanic or Latino205223112116656
Unknown or Not Reported222161362
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Monthly: Pegcetacoplan/PegcetacoplanEOM: Pegcetacoplan/PegcetacoplanMonthly: Sham/PegcetacoplanEOM: Sham/PegcetacoplanTotal
American Indian or Alaska Native00000
Asian30014
Native Hawaiian or Other Pacific Islander00000
Black or African American31004
White221245123128717
More than one race00000
Unknown or Not Reported232261465
08

Study locations

175 sites
  • Retinal Research Institute, LLC
    Phoenix, Arizona 85014, United States
  • Associated Retina Consultants, Ltd.
    Phoenix, Arizona 85020, United States
  • California Retina Consultants
    Bakersfield, California 93309, United States
  • Retinal Diagnostic Center
    Campbell, California 95508, United States
  • The Retina Partners
    Encino, California 91436, United States
  • Retina Consultants of Orange County
    Fullerton, California 92835, United States
  • Atlantis Eyecare
    Huntington Beach, California 92647, United States
  • Jacobs Retina Center, UCSD
    La Jolla, California 92093, United States
  • Doheny Eye Center UCLA
    Los Angeles, California 90033, United States
  • Northern California Retina Vitreous Associates
    Mountain View, California 94040, United States
  • Retina Institute of California Medical Group
    Palm Desert, California 92260, United States
  • Byers Eye Institute, Stanford University
    Palo Alto, California 94303, United States
  • Retina Consultants San Diego
    Poway, California 92064, United States
  • Orange County Retina Medical Group
    Santa Ana, California 92705, United States
  • California Retina Consultants
    Santa Barbara, California 93103, United States
  • Bay Area Retina Associates
    Walnut Creek, California 94598, United States
  • Southwest Retina Research Center, LLC
    Durango, Colorado 81301, United States
  • Danbury Eye Physicians & Surgeons, PC
    Danbury, Connecticut 06810, United States
  • Retina Group of New England - New London
    Waterford, Connecticut 06835, United States
  • Florida Eye Microsurgical Institute, Inc.
    Boynton Beach, Florida 33426, United States
  • Blue Ocean Clinical Research
    Clearwater, Florida 33761, United States
  • Pinnacle Research Institute
    Fort Lauderdale, Florida 33309, United States
  • Retina Health Center
    Fort Myers, Florida 33907, United States
  • National Ophthalmic Research Institute
    Fort Myers, Florida 33912, United States
  • Bascom Palmer Eye Institute
    Miami, Florida 33136, United States
  • Bascom Palmer Eye Institute at Naples
    Naples, Florida 34103, United States
  • Palm Beach Gardens
    Palm Beach Gardens, Florida 33418, United States
  • Retina Specialty Institute
    Pensacola, Florida 32503, United States
  • Eye Associates of Pinellas
    Pinellas Park, Florida 33782, United States
  • East Florida Eye Institute
    Stuart, Florida 34994, United States
  • Retina Associates of Florida
    Tampa, Florida 33609, United States
  • University of South Florida
    Tampa, Florida 33612, United States
  • Center for Retina and Macular Disease
    Winter Haven, Florida 33880, United States
  • Southeast Retina Center, PC
    Augusta, Georgia 30909, United States
  • Georgia Retina, PC
    Marietta, Georgia 30060, United States
  • Gailey Eye Clinic Retina Center
    Bloomington, Illinois 61704, United States
  • Northwestern Feinberg School of Medicine
    Chicago, Illinois 60611, United States
  • Rush University Medical Center
    Chicago, Illinois 60612, United States
  • Midwest Eye Institute
    Indianapolis, Indiana 46290, United States
  • Retina and Vitreous Associates of Kentucky, PSC dba Retina Associates of Kentucky
    Lexington, Kentucky 40509, United States
  • The Retina Care Center
    Baltimore, Maryland 21209, United States
  • Elman Retina Group
    Baltimore, Maryland 21236, United States
  • The Retina Group of Washington
    Chevy Chase, Maryland 20815, United States
  • Cumberland Valley Retina Center
    Hagerstown, Maryland 21740, United States
  • Mid Atlantic Retina Specialist
    Hagerstown, Maryland 21740, United States
  • Ophthalmic Consultants of Boston
    Boston, Massachusetts 02114, United States
  • Tufts Medical Center
    Boston, Massachusetts 02116, United States
  • New England Retina Consultants, PC
    Springfield, Massachusetts 01107, United States
  • Michigan Medicine Kellogg Eye Center
    Ann Arbor, Michigan 48105, United States
  • Associated Retinal Consultants, PC
    Grand Rapids, Michigan 49546, United States
  • Associated Retinal Consultants, PC
    Traverse City, Michigan 49686, United States
  • Vitreoretinal Surgery
    Minneapolis, Minnesota 55435, United States
  • Sierra Eye Associates
    Reno, Nevada 89503, United States
  • Retina Center of NJ, LLC
    Bloomfield, New Jersey 07003, United States
  • NJ Retina
    Teaneck, New Jersey 07666, United States
  • NJ Retina
    Toms River, New Jersey 08755, United States
  • Vision Research Center Eye Associates of NM
    Albuquerque, New Mexico 87109, United States
  • Long Island Vitreoretinal Consultants
    Great Neck, New York 11021, United States
  • Vitreous Retina Macula Consultants of New York
    New York, New York 10022, United States
  • Ophthalmic Consultants of Long Island
    Oceanside, New York 11572, United States
  • Western Carolina Retinal Associates
    Asheville, North Carolina 28803, United States
  • Charlotte Eye Ear Nose and Throat Associates, PS
    Charlotte, North Carolina 28210, United States
  • Duke University, Duke Eye Center
    Durham, North Carolina 27710, United States
  • Graystone Eye
    Hickory, North Carolina 28602, United States
  • Retina Associates of Cleveland, Inc.
    Cleveland, Ohio 44122, United States
  • Retina Associates of Cleveland
    Cleveland, Ohio 44130, United States
  • Cleveland Clinic Cole Eye Institute
    Cleveland, Ohio 44195, United States
  • The Ohio State University
    Columbus, Ohio 43212, United States
  • Retina Associates of Cleveland, Inc.
    Youngstown, Ohio 44505, United States
  • Retina Vitreous Center, PLLC
    Edmond, Oklahoma 73013, United States
  • Oregon Retina LLP
    Eugene, Oregon 97405, United States
  • Retina Northwest, PC
    Portland, Oregon 97221, United States
  • Eye Health Northwest
    Portland, Oregon 97225, United States
  • Mid Atlantic Retina
    Bethlehem, Pennsylvania 18107, United States
  • Retina Vitreous Consultants
    Monroeville, Pennsylvania 15146, United States
  • Mid Atlantic Retina
    Philadelphia, Pennsylvania 19006, United States
  • Mid Atlantic Retina
    Philadelphia, Pennsylvania 19107, United States
  • AIO Visionary Eye Care
    West Mifflin, Pennsylvania 15122, United States
  • Black Hills Regional Eye Institute
    Rapid City, South Dakota 57701, United States
  • Charles Retina Institute
    Germantown, Tennessee 38138, United States
  • Tennessee Retina, PC
    Nashville, Tennessee 78503, United States
  • Retina Research Center PLLC
    Austin, Texas 78750, United States
  • Brown Retina Institute
    Bellaire, Texas 77401, United States
  • Texas Retina Associates
    Dallas, Texas 75231, United States
  • Houston Eye Associates
    Houston, Texas 77025, United States
  • Retina Consultants of Houston, PA
    Houston, Texas 77030, United States
  • Valley Retina Institute
    McAllen, Texas 78503, United States
  • Medical Center Ophthalmology Associates
    San Antonio, Texas 78240, United States
  • Retina Associates of South Texas
    San Antonio, Texas 78240, United States
  • Retina Consultants of Houston
    The Woodlands, Texas 77384, United States
  • Retina Associates of Utah, PC
    Murray, Utah 84107, United States
  • University of Utah - John A. Moran Center
    Salt Lake City, Utah 84312, United States
  • University of Virginia
    Charlottesville, Virginia 22903, United States
  • The Retina Group of Washington
    Fairfax, Virginia 22031, United States
  • Emerson Clinical Research Institute
    Falls Church, Virginia 22046, United States
  • Vitreoretinal Associates of Washington
    Bellevue, Washington 98004, United States
  • Spokane Eye Clinical Research
    Spokane, Washington 99204, United States
  • Retina Health Center
    Madison, Wisconsin 53705, United States
  • Retina-Vitreous Associates Medical Group
    Madison, Wisconsin 53705, United States
  • Diagnostico Ocular
    Buenos Aires, 1425, Argentina

Showing the first 100 of 175 sites across 15 countries. Choose a country and city above to see the rest.

09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Mar 2021 to primary completion in Sep 2025, 54 months.

Results were first posted in Oct 2026, 1 year 1 month after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 23, 2021
First posted
Feb 25, 2021
StartActual
Mar 4, 2021
Primary completionActual
Sep 5, 2025
CompletionActual
Sep 5, 2025
Results first posted
Oct 28, 2026
Last update
Oct 28, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Results
Results posted
posted Oct 28, 2026
Sites
4 sites added, 4 sites removed
Show 4 added (3 United States, 1 United Kingdom)
  • Retinal Research Institute, LLC · Phoenix, United States
  • Pinnacle Research Institute · Fort Lauderdale, United States
  • National Ophthalmic Research Institute · Fort Myers, United States
  • Kings College Hospital NHS Trust · London, United Kingdom
Show 4 removed
  • Retinal Research Institue, LLC · Phoenix, United States
  • Pinnacle Research Insitute · Fort Lauderdale, United States
  • National Opthalmic Research Institute · Fort Myers, United States
  • Kings College Hospital NHS Trust · London, United Kingdom
Oct 28, 2026
Eligibility
1 exclusion criterion edited
What changed in the criteria
Exclusion criteria
  • EditedIf, 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 yearsoveryears over the next 36 months.
Oct 28, 2026
Interventions
Arms added: Monthly: Pegcetacoplan/ Pegcetacoplan·EOM: Pegcetacoplan/Pegcetacoplan·Monthly: Sham/Pegcetacoplan·EOM: Sham/Pegcetacoplan; arms removed: Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months·Pegcetacoplan, 15 mg/100 μL, every other month (EOM) for up to 36 months
Oct 28, 2026
Primary outcomes
1 edited
What changed in the outcomes
  • ChangedBefore Incidence and severity of ocular and systemic adverse eventsNow Number of Participants Who Experienced at Least One Ocular and Systemic Adverse Events (AEs)Time frame: Up to 36 Months → From Day 1 up to Month 36
Oct 28, 2026
Documents
Oct 28, 2026
Secondary outcomes
3 edited, 14 added
What changed in the outcomes
  • EditedChange from baselineFrom Baseline in the total areaTotal Area of Geographic Atrophy (GA lesion) Lesion(s) in the study eye (in mm2)Study EyeTime frame: Up to 36 Months → Baseline, Months 12, 24 and 36
  • EditedChange from baselineFrom Baseline in Normal-Luminance Best-Corrected Visual Acuity (NL-BCVA score (study eye)) Score of the Study EyeTime frame: Up to 36 Months → Baseline, Months 12, 24 and 36
  • EditedChange from baselineFrom Baseline in Low-Luminance Best-Corrected Visual Acuity (LL-BCVA score (study eye)) Score of the Study EyeTime frame: Up to 36 Months → Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in the Rate of GA Lesion Growth in the Study EyeTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in Monocular Maximum Corrected Reading Speed of the Study EyeTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in Binocular Maximum Corrected Reading SpeedTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in Number of Scotomatous Points of the Study EyeTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in Macular Sensitivity of the Study EyeTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in 95% Bivariate Contour Ellipse Area (BCEA) Degrees Squared (Deg²) of the Study EyeTime frame: Baseline, Months 12, 24 and 36
  • AddedMean Change From Baseline in Functional Reading Independence (FRI) Index ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) Composite ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in NEI VFQ-25 Near Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From APL2-GA-305 Baseline in NEI VFQ-25 Distance Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in NEI VFQ-25 Driving Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in National Eye Institute Visual Functioning Questionnaire 39-Item Version (NEI VFQ-39) Composite ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in NEI VFQ-39 Near Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
  • AddedChange From Baseline in NEI VFQ-39 Distance Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
Oct 28, 2026
Show all 1 update
  1. Oct 28, 2026
    Results posted
    Documents Study protocol ↗ posted · Statistical analysis plan ↗ posted
    4 sites added, 4 sites removed
    Show 4 added (3 United States, 1 United Kingdom)
    • Retinal Research Institute, LLC · Phoenix, United States
    • Pinnacle Research Institute · Fort Lauderdale, United States
    • National Ophthalmic Research Institute · Fort Myers, United States
    • Kings College Hospital NHS Trust · London, United Kingdom
    Show 4 removed
    • Retinal Research Institue, LLC · Phoenix, United States
    • Pinnacle Research Insitute · Fort Lauderdale, United States
    • National Opthalmic Research Institute · Fort Myers, United States
    • Kings College Hospital NHS Trust · London, United Kingdom
    Eligibility 1 exclusion criterion edited
    What changed in the criteria
    Exclusion criteria
    • EditedIf, 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 yearsoveryears over the next 36 months.
    Primary outcomes 1 edited
    What changed in the outcomes
    • ChangedBefore Incidence and severity of ocular and systemic adverse eventsNow Number of Participants Who Experienced at Least One Ocular and Systemic Adverse Events (AEs)Time frame: Up to 36 Months → From Day 1 up to Month 36
    Secondary outcomes 3 edited, 14 added
    What changed in the outcomes
    • EditedChange from baselineFrom Baseline in the total areaTotal Area of Geographic Atrophy (GA lesion) Lesion(s) in the study eye (in mm2)Study EyeTime frame: Up to 36 Months → Baseline, Months 12, 24 and 36
    • EditedChange from baselineFrom Baseline in Normal-Luminance Best-Corrected Visual Acuity (NL-BCVA score (study eye)) Score of the Study EyeTime frame: Up to 36 Months → Baseline, Months 12, 24 and 36
    • EditedChange from baselineFrom Baseline in Low-Luminance Best-Corrected Visual Acuity (LL-BCVA score (study eye)) Score of the Study EyeTime frame: Up to 36 Months → Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in the Rate of GA Lesion Growth in the Study EyeTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in Monocular Maximum Corrected Reading Speed of the Study EyeTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in Binocular Maximum Corrected Reading SpeedTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in Number of Scotomatous Points of the Study EyeTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in Macular Sensitivity of the Study EyeTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in 95% Bivariate Contour Ellipse Area (BCEA) Degrees Squared (Deg²) of the Study EyeTime frame: Baseline, Months 12, 24 and 36
    • AddedMean Change From Baseline in Functional Reading Independence (FRI) Index ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) Composite ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in NEI VFQ-25 Near Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From APL2-GA-305 Baseline in NEI VFQ-25 Distance Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in NEI VFQ-25 Driving Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in National Eye Institute Visual Functioning Questionnaire 39-Item Version (NEI VFQ-39) Composite ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in NEI VFQ-39 Near Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
    • AddedChange From Baseline in NEI VFQ-39 Distance Activity Subscale ScoreTime frame: Baseline, Months 12, 24 and 36
    Interventions Arms added: Monthly: Pegcetacoplan/ Pegcetacoplan·EOM: Pegcetacoplan/Pegcetacoplan·Monthly: Sham/Pegcetacoplan·EOM: Sham/Pegcetacoplan; arms removed: Pegcetacoplan, 15 mg/100 μL, monthly for up to 36 months·Pegcetacoplan, 15 mg/100 μL, every other month (EOM) for up to 36 months
    + 2 other changes: index terms and verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

11

Registry details

Key details

Study ID
NCT04770545
Lead sponsor
Apellis Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Feb 25, 2021
Start date
Mar 4, 2021
Primary completion
Sep 5, 2025
Completion
Sep 5, 2025
Results posted
Oct 28, 2026
Last update
Oct 28, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Pegcetacoplan

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