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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02461771




Registration number
NCT02461771
Ethics application status
Date submitted
2/06/2015
Date registered
3/06/2015
Date last updated
6/10/2020

Titles & IDs
Public title
Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD
Scientific title
Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal APL-2 Therapy for Neovascular Age-Related Macular Degeneration (AMD)
Secondary ID [1] 0 0
POT-CP043014
Universal Trial Number (UTN)
Trial acronym
ASAP II
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neovascular Age-Related Macular Degeneration 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan

Experimental: Pegcetacoplan Cohort 1 - 4 mg of pegcetacoplan 100 µL IVT injection

Experimental: Pegcetacoplan Cohort 2 - 10 mg of pegcetacoplan 100 µL IVT injection

Experimental: Pegcetacoplan Cohort 3 - 20 mg of pegcetacoplan 100 µL IVT injection


Treatment: Drugs: Pegcetacoplan
On treatment day, subjects will be administered a single 100 µL IVT injection of pegcetacoplan at the dose corresponding to their treatment assignment.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Subjects Who Experienced Ocular and Systemic Adverse Events (AEs), Including by Severity
Timepoint [1] 0 0
Day 1 to Day 113
Primary outcome [2] 0 0
Number of Dose Limiting Toxicities (DLTs)
Timepoint [2] 0 0
Day 1 to Day 15
Primary outcome [3] 0 0
Median Area Under the Serum Concentration-Time Curve From Time Zero to Last Measurable Concentration (AUC[0-t])
Timepoint [3] 0 0
Predose (screening), postdose Day 3 to Day 113
Primary outcome [4] 0 0
Median Dose Normalized AUC(0-t)
Timepoint [4] 0 0
Predose (screening), postdose Day 3 to Day 113
Primary outcome [5] 0 0
Maximum Observed Serum Concentration (Cmax)
Timepoint [5] 0 0
Predose (screening), postdose Day 3 to Day 113
Primary outcome [6] 0 0
Median Dose Normalized Cmax
Timepoint [6] 0 0
Predose (screening), postdose Day 3 to Day 113
Primary outcome [7] 0 0
Median Time to the Maximum Measured Serum Concentration (Tmax)
Timepoint [7] 0 0
Predose (screening), postdose Day 3 to Day 113
Secondary outcome [1] 0 0
Median Change From Baseline in Visual Acuity for the Study Eye
Timepoint [1] 0 0
Day 1 to Day 113
Secondary outcome [2] 0 0
Median Change From Baseline in Central Retinal Thickness, Central Retinal Lesion Thickness and Central Subfield Thickness in the Study Eye
Timepoint [2] 0 0
Day 1 to Day 113
Secondary outcome [3] 0 0
Median Change From Baseline in Macular Cube Volume in the Study Eye
Timepoint [3] 0 0
Day 1 to Day 113

Eligibility
Key inclusion criteria
1. Male or Female
2. Age = 50 years
3. The presence of an active choroidal neovascular lesion secondary to AMD
4. On treatment with anti-VEGF therapy (Lucentis®, Eylea® or Avastin®)
5. Must have received at least 3 anti-VEGF treatments over the 26-week period prior to screening (Screening Visit)
6. Evidence that the macular fluid has responded to anti-VEGF in the past based on OCT in the opinion of PI
7. At screening, evidence of subretinal fluid and retinal cystic changes
8. Must have received anti-VEGF treatment within 10 days prior to pegcetacoplan treatment (anti-VEGF can be administered on the same day of the screening visit after the screening procedures have been completed)
9. OCTs of sufficient quality to allow for the assessment of the central macular fluid can be obtained
10. Female subjects must be:

* Women of non-child-bearing potential (WONCBP), Or
* 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
11. 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
12. Willing and able to give informed consent
Minimum age
50 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Choroidal neovascularization associated with other ocular diseases such as pathologic myopia, ocular histoplasmosis or posterior uveitis, etc
2. Decreased vision due to retinal disease not attributable to choroidal neovascularization, such as nonexudative forms of AMD, geographic atrophy, inherited retinal dystrophy, uveitis or epiretinal membrane, a vitelliform-like lesion of the outer retina (e.g., as in pattern dystrophies or basal laminar drusen), idiopathic parafoveal telangiectasis, or central serous retinopathy
3. Additional ocular diseases that have irreversibly compromised or, during follow-up, could likely compromise the VA of the study eye including amblyopia, anterior ischemic optic neuropathy, clinically significant diabetic macular edema, severe non proliferative diabetic retinopathy, or proliferative diabetic retinopathy
4. Decreased vision due to significant media opacity such as corneal disease or cataract, or opacity precluding photography of the retina
5. Cataract surgery within three months of enrollment
6. Presence of any hemorrhage
7. History of treatment for CNV:

1. Previous PDT treatment within 30 days prior to enrollment in the study
2. Previous extrafoveal or juxtafoveal thermal laser photocoagulation within 30 days prior to enrollment in the study
8. Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization
9. Medical problems that make consistent follow-up over the treatment period unlikely (e.g. stroke, severe MI, end stage malignancy), or in general a poor medical risk because of other systemic diseases or active uncontrolled infections
10. Hypersensitivity to fluorescein

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
Other
Other design features
Phase
Phase 1
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
28/01/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/03/2016
Sample size
Target
Accrual to date
Final
13
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Australia, New South Wells - Parramatta
Recruitment postcode(s) [1] 0 0
2150 - Parramatta
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
New Hampshire

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

Ethics approval
Ethics application status

Summary
Brief summary
The objective of this study is to provide initial safety, tolerability and pharmacokinetics information of intravitreal administration of pegcetacoplan in order to support further development into larger Phase II studies for treatment of patients with AMD.
Trial website
https://clinicaltrials.gov/study/NCT02461771
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02461771