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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02503332
Registration number
NCT02503332
Ethics application status
Date submitted
14/07/2015
Date registered
20/07/2015
Date last updated
6/10/2020
Titles & IDs
Public title
Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy
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Scientific title
A Phase II, Multicenter, Randomized, Single-Masked, Sham-Controlled Study of Safety, Tolerability and Evidence of Activity of Intravitreal APL-2 Therapy in Patients With Geographic Atrophy (GA)
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Secondary ID [1]
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POT-CP121614
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Universal Trial Number (UTN)
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Trial acronym
FILLY
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy
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Condition category
Condition code
Eye
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Diseases / disorders of the eye
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan
Other interventions - Sham Procedure
Experimental: Pegcetacoplan 15 mg/100 µL Monthly for 12 months - A single dose of 15 mg pegcetacoplan/100 µL will be administered via intravitreal injection in this study. Subjects will receive an injection every month for 12 consecutive months.
Experimental: Pegcetacoplan 15 mg/100 µL EOM for 12 months - 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 (EOM) for 12 consecutive months.
Sham Comparator: Sham Monthly for 12 months - Subjects will receive a Sham procedure every month for 12 consecutive months.
Sham Comparator: Sham EOM for 12 months - Subjects will receive a Sham procedure every other month (EOM) for 12 consecutive months.
Treatment: Drugs: Pegcetacoplan
Other interventions: Sham Procedure
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Least Square (LS) Mean Change From Baseline in Square Root GA Lesion Size in the Study Eye at Month 12
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Assessment method [1]
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The square root GA lesion size (i.e. transformed area of GA) was measured by FAF photographs. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [1]
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Baseline (screening) and Month 12.
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Primary outcome [2]
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Number of Subjects With Treatment Emergent Adverse Events (TEAEs) in the Study Eye, Including by Severity
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Assessment method [2]
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A TEAE was defined as any adverse event (AE) that commenced or worsened on or after time of first study drug administration up to 60 days beyond last dose of study drug. A treatment-related TEAE was defined as a TEAE with a relationship to study drug of possibly related or probably related or not reported. Severity of TEAEs were categorized as mild; moderate; severe; life-threatening or death related to TEAE, according to Common Terminology Criteria for AEs v4.03. A TEAE of special interest (TEAESI) was defined as a TEAE of scientific and medical concern specific to pegcetacoplan, whether serious or non-serious.
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Timepoint [2]
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From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).
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Secondary outcome [1]
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LS Mean Change From Baseline in Untransformed GA Lesion Size in the Study Eye at Month 12
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Assessment method [1]
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The untransformed area of GA was measured by FAF. Baseline is defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [1]
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Baseline (Day 1) and Month 12.
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Secondary outcome [2]
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LS Mean Change From Baseline in Best-Corrected Visual Acuity (BCVA) Score of the Study Eye at Month 12
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Assessment method [2]
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The BCVA letter score was determined using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart assessed at a starting distance of 4 meters. The score ranges from 0 to 100 letters, lower number indicating reduced visual acuity; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.
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Timepoint [2]
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Baseline (Day 1) and Month 12.
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Secondary outcome [3]
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LS Mean Change From Baseline in Low Luminance BCVA (LL-BCVA) Score in the Study Eye at Month 12
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Assessment method [3]
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The LL-BCVA was measured by placing a 2.0-log-unit neutral density filter over the best correction and having the participant read the normally illuminated ETDRS chart. The score ranges from 0 to 100 letters, lower number indicating worse vision; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.
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Timepoint [3]
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Baseline (Day 1) and Month 12.
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Secondary outcome [4]
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LS Mean Change From Baseline in Low Luminance VA (LL-VA) Deficit Score in the Study Eye at Month 12
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Assessment method [4]
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The LL-VA deficit score is calculated as BCVA score minus LL-BCVA score. The LL-VA deficit score ranges from 0 to 100 letters, lower number indicating worse deficit.
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Timepoint [4]
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Baseline (Day 1) and Month 12.
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Secondary outcome [5]
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LS Mean Change From Baseline in Distance of GA Lesion From the Fovea (Foveal Encroachment) in the Study Eye at Month 12
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Assessment method [5]
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The foveal encroachment in the study eye was measured by FAF. Baseline is defined as the last available, non-missing observation prior to first study drug administration.
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Timepoint [5]
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Baseline (Day 1) and Month 12.
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Secondary outcome [6]
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Number of Subjects With Any Macular Neovascularization (MNV) TEAEs in the Study Eye
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Assessment method [6]
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The number of subjects with any MNV TEAEs in the study eye was identified via clinical review of all ocular TEAEs.
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Timepoint [6]
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From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).
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Eligibility
Key inclusion criteria
Unless specified otherwise, ocular specific inclusion criteria apply to
the study eye only.
1. Male or Female.
2. Age greater than or equal to 50 years.
3. BCVA of 20/320 (Snellen equivalent) or better using ETDRS charts.
4. Diagnosis of GA of the macula secondary to age-related macular degeneration, confirmed
within 14 days prior to randomization by the central reading center (CRC) using Fundus
Autofluorescence (FAF) images, as well as the following criteria:
1. Total GA area must be = 2.5 and = 17.5 mm2 (1 and 7 disk areas [DA]
respectively), determined by screening images of FAF.
2. If GA is multifocal, at least one focal lesion must be = 1.25 mm2 (0.5 DA).
3. GA can be completely visualized on the macula centered image.
4. GA must be able to be photographed in its entirety.
5. GA must be able to be measured separately from any areas of peripapillary atrophy
as assessed by the CRC.
6. Presence of any pattern of hyperautofluorescence in the junctional zone of GA.
Absence of hyperautoflouorescence (i.e. pattern = none) is exclusionary. See Holz
et al. 2007.1
5. Female subjects must be:
1. Women of non-child-bearing potential (WONCBP), or
2. Women of child-bearing potential (WOCBP) with a negative pregnancy test at
screening and must agree to use protocol defined methods of contraception for the
duration of the study.
6. 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.
7. Willing and able to give informed consent.
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Minimum age
50
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Unless specified otherwise, ocular specific inclusion criteria apply to
the study eye only.
1. GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy or toxic
maculopathies like plaquenil maculopathy.
2. Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or
an axial length >26 mm.
3. Any history or current evidence of exudative ("wet") AMD including any evidence of
retinal pigment epithelium rips or evidence of neovascularization anywhere in the
retina based on fluorescein angiogram as assessed by the CRC.
4. Retinal disease other than AMD; however, benign conditions of the vitreous or
peripheral retina are not exclusionary (i.e. pavingstone degeneration).
5. Any ophthalmologic condition that reduces the clarity of the media and that, in the
opinion of the Investigator interferes with ophthalmologic examination (e.g. advanced
cataract or corneal abnormalities).
6. Any ophthalmologic condition that prevents adequate imaging of the retina judged by
the site or CRC.
7. Intraocular surgery (including lens replacement surgery) within 3 months prior to
randomization.
8. Aphakia or absence of the posterior capsule. Previous violation of the posterior
capsule is also excluded unless it occurred as a result of yttrium aluminum garnet
(YAG) laser posterior capsulotomy in association with prior posterior chamber
intraocular lens implantation and at least 60 days prior to Day 0.
9. Any ophthalmic condition that may require surgery during the study period.
10. Any contraindication to IVT injection including current ocular or periocular
infection.
11. History of uveitis or endophthalmitis.
12. History of IVT injection at any time.
13. Participation in another interventional clinical study, or use of any experimental
treatment for AMD or any other investigational new drug within 6 weeks or 5 half-lives
of the active (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.
14. Medical or psychiatric conditions that, in the opinion of the investigator, make
consistent follow-up over the treatment period unlikely, or in general a poor medical
risk because of other systemic diseases or active uncontrolled infections.
15. 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.
16. Hypersensitivity to fluorescein.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/09/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
17/01/2018
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Sample size
Target
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Accrual to date
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Final
246
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Recruitment in Australia
Recruitment state(s)
NSW,TAS,VIC,WA
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Recruitment hospital [1]
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Marsden Eye Specialists - Paramatta
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Recruitment hospital [2]
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Save Sight Institute, Sydney Eye Hospital - Sydney
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Recruitment hospital [3]
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Sydney Retina Clinic and Day Surgery - Sydney
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Recruitment hospital [4]
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Sydney West Retina - Westmead
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Hobart eye Surgeons - Hobart
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Recruitment hospital [6]
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Tasmanian Eye Institute - South Launceston
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Recruitment hospital [7]
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Royal Victorian Eye and Ear Hospital - East Melbourne
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Recruitment hospital [8]
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Center for Eye Research Australia - Melbourne
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Recruitment hospital [9]
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Lions Eye Institute - Nedlands
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Recruitment postcode(s) [1]
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2150 - Paramatta
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Recruitment postcode(s) [2]
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2000 - Sydney
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Recruitment postcode(s) [3]
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2145 - Westmead
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Recruitment postcode(s) [4]
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7002 - Hobart
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Recruitment postcode(s) [5]
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7249 - South Launceston
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Recruitment postcode(s) [6]
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3002 - East Melbourne
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Recruitment postcode(s) [7]
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3002 - Melbourne
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Recruitment postcode(s) [8]
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6009 - Nedlands
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Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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California
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Connecticut
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Florida
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Georgia
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Auckland
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New Zealand
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Christchurch
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Apellis Pharmaceuticals, Inc.
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objectives of the study are to assess the safety, tolerability and evidence of
activity of multiple intravitreal (IVT) injections of pegcetacoplan in subjects with
Geographic Atrophy associated with Age-Related Macular Degeneration (AMD).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02503332
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Federico Grossi, MD PhD
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Address
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Apellis Pharmaceuticals, Inc.
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02503332
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