Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy
Condition: Geographic Atrophy
MeSH terms: Geographic Atrophy; Atrophy
Intervention: Pegcetacoplan; Sham Procedure
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: Single (Participant)
Enrollment: 246.0
Sex: All
Minimum age: 50 Years
Healthy volunteers: No

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).
    

Eligibility criteria:

        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.

        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.