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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/NCT04770545




Registration number
NCT04770545
Ethics application status
Date submitted
23/02/2021
Date registered
25/02/2021
Date last updated
31/08/2023

Titles & IDs
Public title
An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD
Scientific title
A Phase 3, Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration
Secondary ID [1] 0 0
APL2-GA-305
Universal Trial Number (UTN)
Trial acronym
GALE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy Secondary to Age-related Macular Degeneration 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 - PEGCETACOPLAN (APL-2)

Experimental: Pegcetacoplan, 15 mg/100 µL, monthly for up to 36 months - Participants from Study APL2-103 (NCT03777332) or those who completed the treatment at Month 24 from either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600) and were administered monthly intravitreal (IVT) pegcetacoplan (15 mg/100 µL) or monthly sham will receive IVT pegcetacoplan (15 mg/100 µL) monthly for up to approximately 36 months.

Experimental: Pegcetacoplan, 15 mg/100 µL, every other month (EOM) for up to 36 months - Participants from Study APL2-103 (NCT03777332) or those who completed the treatment at Month 24 from either Study APL2-303 (Derby, NCT03525613) or Study APL2-304 (Oaks, NCT03525600) and were administered every other month (EOM) intravitreal (IVT) pegcetacoplan (15 mg/100 µL) or EOM sham will receive IVT pegcetacoplan (15 mg/100 µL) EOM for up to approximately 36 months.


Treatment: Drugs: PEGCETACOPLAN (APL-2)
Complement (C3) Inhibitor
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence and severity of ocular and systemic adverse events
Timepoint [1] 0 0
Up to 36 Months
Secondary outcome [1] 0 0
Change from baseline in the total area of GA lesion(s) in the study eye (in mm2)
Timepoint [1] 0 0
Up to 36 Months
Secondary outcome [2] 0 0
Change from baseline in NL-BCVA score (study eye)
Timepoint [2] 0 0
Up to 36 Months
Secondary outcome [3] 0 0
Change from baseline in LL-BCVA score (study eye)
Timepoint [3] 0 0
Up to 36 Months

Eligibility
Key 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.
Minimum age
60 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 yearsover 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2025
Actual
Sample size
Target
1200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Marsden Eye Specialist - Parramatta
Recruitment hospital [2] 0 0
Strathfield Retina Clinic - Strathfield
Recruitment hospital [3] 0 0
Sydney Retina - Sydney
Recruitment hospital [4] 0 0
The Ashley Centre - Westmead
Recruitment hospital [5] 0 0
Centre for Eye Research Australia - East Melbourne
Recruitment hospital [6] 0 0
Retina and Eye Consultantss - Hurstville
Recruitment hospital [7] 0 0
Lions Eye Institute - Nedlands
Recruitment hospital [8] 0 0
Save Sight Institute - Westmead
Recruitment postcode(s) [1] 0 0
2150 - Parramatta
Recruitment postcode(s) [2] 0 0
2135 - Strathfield
Recruitment postcode(s) [3] 0 0
2000 - Sydney
Recruitment postcode(s) [4] 0 0
2145 - Westmead
Recruitment postcode(s) [5] 0 0
3002 - East Melbourne
Recruitment postcode(s) [6] 0 0
2220 - Hurstville
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Apellis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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).
Trial website
https://clinicaltrials.gov/ct2/show/NCT04770545
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
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Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04770545