Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD
Condition: Neovascular Age-Related Macular Degeneration
MeSH terms: Macular Degeneration; Wet Macular Degeneration
Intervention: Pegcetacoplan
Intervention description: On treatment day, subjects will be administered a single 100 μL IVT injection of pegcetacoplan at the dose corresponding to their treatment assignment.
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Sequential Assignment
Masking: None (Open Label)
Enrollment: 13.0
Sex: All
Minimum age: 50 Years
Healthy volunteers: No
Keywords: AMD; Wet AMD; Neovascular AMD

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.
    

Eligibility criteria:

        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

        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