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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02503332




Registration number
NCT02503332
Ethics application status
Date submitted
14/07/2015
Date registered
20/07/2015
Date last updated
19/11/2019

Titles & IDs
Public title
Study of of APL-2 Therapy in Patients Geographic Atrophy
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 Geographic Atrophy (GA)
Secondary ID [1] 0 0
POT-CP121614
Universal Trial Number (UTN)
Trial acronym
FILLY
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - APL-2
Other interventions - Sham Procedure

Experimental: APL-2 15 mg/100 µL Monthly for 12 months - A single dose of 15 mg APL-2/100 µL will be administered via intravitreal injection in this study. Subjects will receive an injection every month for 12 consecutive months.

Experimental: APL-2 15 mg/100 µL EOM for 12 months - A single dose of 15 mg APL-2/100 µL will be administered via intravitreal injection in this study. Subjects will receive an injections 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: APL-2


Other interventions: Sham Procedure


Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Primary Efficacy Endpoint: The primary endpoint is the change in square root geographic atrophy (GA) lesion size from baseline at month 12 as measured by FAF.
Timepoint [1] 0 0
12 months from baseline
Primary outcome [2] 0 0
Primary Safety Endpoint: Number of local and systemic treatment emergent averse events (TEAE).
Timepoint [2] 0 0
12 months from baseline
Primary outcome [3] 0 0
Primary Safety Endpoint: Severity of local and systemic treatment emergent averse events (TEAE).
Timepoint [3] 0 0
12 months from baseline

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 of 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.
Minimum age
50 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,TAS,VIC,WA
Recruitment hospital [1] 0 0
Marsden Eye Specialists - Paramatta
Recruitment hospital [2] 0 0
Save Sight Institute, Sydney Eye Hospital - Sydney
Recruitment hospital [3] 0 0
Sydney Retina Clinic and Day Surgery - Sydney
Recruitment hospital [4] 0 0
Sydney West Retina - Westmead
Recruitment hospital [5] 0 0
Hobart eye Surgeons - Hobart
Recruitment hospital [6] 0 0
Tasmanian Eye Institute - South Launceston
Recruitment hospital [7] 0 0
Royal Victorian Eye and Ear Hospital - East Melbourne
Recruitment hospital [8] 0 0
Lions Eye Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2150 - Paramatta
Recruitment postcode(s) [2] 0 0
2000 - Sydney
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment postcode(s) [4] 0 0
7002 - Hobart
Recruitment postcode(s) [5] 0 0
7249 - South Launceston
Recruitment postcode(s) [6] 0 0
3002 - East Melbourne
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
New Hampshire
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
North Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Tennessee
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
New Zealand
State/province [15] 0 0
Auckland
Country [16] 0 0
New Zealand
State/province [16] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Apellis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

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 APL-2 in subjects with Geographic
Atrophy associated with Age-Related Macular Degeneration (AMD).
Trial website
https://clinicaltrials.gov/show/NCT02503332
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Federico Grossi, MD PhD
Address 0 0
Apellis Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications