Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
Query!
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)
Query!
Secondary ID [1]
0
0
POT-CP121614
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
FILLY
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy
0
0
Query!
Condition category
Condition code
Eye
0
0
0
0
Query!
Diseases / disorders of the eye
Query!
Other
0
0
0
0
Query!
Research that is not of generic health relevance and not applicable to specific health categories listed above
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
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.
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Least Square (LS) Mean Change From Baseline in Square Root GA Lesion Size in the Study Eye at Month 12
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline (screening) and Month 12.
Query!
Primary outcome [2]
0
0
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) in the Study Eye, Including by Severity
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).
Query!
Secondary outcome [1]
0
0
LS Mean Change From Baseline in Untransformed GA Lesion Size in the Study Eye at Month 12
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline (Day 1) and Month 12.
Query!
Secondary outcome [2]
0
0
LS Mean Change From Baseline in Best-Corrected Visual Acuity (BCVA) Score of the Study Eye at Month 12
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Baseline (Day 1) and Month 12.
Query!
Secondary outcome [3]
0
0
LS Mean Change From Baseline in Low Luminance BCVA (LL-BCVA) Score in the Study Eye at Month 12
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
Baseline (Day 1) and Month 12.
Query!
Secondary outcome [4]
0
0
LS Mean Change From Baseline in Low Luminance VA (LL-VA) Deficit Score in the Study Eye at Month 12
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Baseline (Day 1) and Month 12.
Query!
Secondary outcome [5]
0
0
LS Mean Change From Baseline in Distance of GA Lesion From the Fovea (Foveal Encroachment) in the Study Eye at Month 12
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline (Day 1) and Month 12.
Query!
Secondary outcome [6]
0
0
Number of Subjects With Any Macular Neovascularization (MNV) TEAEs in the Study Eye
Query!
Assessment method [6]
0
0
The number of subjects with any MNV TEAEs in the study eye was identified via clinical review of all ocular TEAEs.
Query!
Timepoint [6]
0
0
From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).
Query!
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.
Query!
Minimum age
50
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
24/09/2015
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
17/01/2018
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
246
Query!
Recruitment in Australia
Recruitment state(s)
NSW,TAS,VIC,WA
Query!
Recruitment hospital [1]
0
0
Marsden Eye Specialists - Paramatta
Query!
Recruitment hospital [2]
0
0
Save Sight Institute, Sydney Eye Hospital - Sydney
Query!
Recruitment hospital [3]
0
0
Sydney Retina Clinic and Day Surgery - Sydney
Query!
Recruitment hospital [4]
0
0
Sydney West Retina - Westmead
Query!
Recruitment hospital [5]
0
0
Hobart eye Surgeons - Hobart
Query!
Recruitment hospital [6]
0
0
Tasmanian Eye Institute - South Launceston
Query!
Recruitment hospital [7]
0
0
Royal Victorian Eye and Ear Hospital - East Melbourne
Query!
Recruitment hospital [8]
0
0
Center for Eye Research Australia - Melbourne
Query!
Recruitment hospital [9]
0
0
Lions Eye Institute - Nedlands
Query!
Recruitment postcode(s) [1]
0
0
2150 - Paramatta
Query!
Recruitment postcode(s) [2]
0
0
2000 - Sydney
Query!
Recruitment postcode(s) [3]
0
0
2145 - Westmead
Query!
Recruitment postcode(s) [4]
0
0
7002 - Hobart
Query!
Recruitment postcode(s) [5]
0
0
7249 - South Launceston
Query!
Recruitment postcode(s) [6]
0
0
3002 - East Melbourne
Query!
Recruitment postcode(s) [7]
0
0
3002 - Melbourne
Query!
Recruitment postcode(s) [8]
0
0
6009 - Nedlands
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Alabama
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Arizona
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
California
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Connecticut
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Florida
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Georgia
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Illinois
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Indiana
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Maryland
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Massachusetts
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Michigan
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Minnesota
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
New Hampshire
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
New York
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
North Carolina
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Ohio
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Pennsylvania
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
South Dakota
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
Tennessee
Query!
Country [20]
0
0
United States of America
Query!
State/province [20]
0
0
Texas
Query!
Country [21]
0
0
United States of America
Query!
State/province [21]
0
0
Utah
Query!
Country [22]
0
0
New Zealand
Query!
State/province [22]
0
0
Auckland
Query!
Country [23]
0
0
New Zealand
Query!
State/province [23]
0
0
Christchurch
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Apellis Pharmaceuticals, Inc.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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).
Query!
Trial website
https://clinicaltrials.gov/study/NCT02503332
Query!
Trial related presentations / publications
Vogl WD, Riedl S, Mai J, Reiter GS, Lachinov D, Bogunovic H, Schmidt-Erfurth U. Predicting Topographic Disease Progression and Treatment Response of Pegcetacoplan in Geographic Atrophy Quantified by Deep Learning. Ophthalmol Retina. 2023 Jan;7(1):4-13. doi: 10.1016/j.oret.2022.08.003. Epub 2022 Aug 7. Liao DS, Metlapally R, Joshi P. Pegcetacoplan treatment for geographic atrophy due to age-related macular degeneration: a plain language summary of the FILLY study. Immunotherapy. 2022 Sep;14(13):995-1006. doi: 10.2217/imt-2022-0078. Epub 2022 Jul 21. Nittala MG, Metlapally R, Ip M, Chakravarthy U, Holz FG, Staurenghi G, Waheed N, Velaga SB, Lindenberg S, Karamat A, Koester J, Ribeiro R, Sadda S. Association of Pegcetacoplan With Progression of Incomplete Retinal Pigment Epithelium and Outer Retinal Atrophy in Age-Related Macular Degeneration: A Post Hoc Analysis of the FILLY Randomized Clinical Trial. JAMA Ophthalmol. 2022 Mar 1;140(3):243-249. doi: 10.1001/jamaophthalmol.2021.6067.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Federico Grossi, MD PhD
Query!
Address
0
0
Apellis Pharmaceuticals, Inc.
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/32/NCT02503332/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/32/NCT02503332/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02503332
Download to PDF