Geographic Atrophy Secondary to Age-Related Macular Degeneration MedDRA version: 20.1 Level: LLT Classification code 10063947 Term: Geographic atrophy System Organ Class: 100000004853
Conditions
Sponsors
Apellis Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age = 60 years. 2. NL-BCVA of 24 letters or better using ETDRS charts. 3. Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center. 4. The GA lesion must meet the criteria as determined by the central reading center's assessment of FAF imaging at screening. 5. Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the Investigator. 6. Female subjects must be women of non-child-bearing potential or women of child-bearing potential with a negative pregnancy test at screening and must agree to use protocol defined methods of contraception. 7. Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception 8. Willing and able to give informed consent and to comply with the study procedures and assessments. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 550
Exclusion criteria
Exclusion criteria: 1. GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye. 2. Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm. 3. Any history or active CNV, associated with AMD or any other cause, including any evidence of RPE rips or evidence of neovascularization anywhere based on spectral domain optical coherence tomography imaging and/or FA as assessed by the Reading Center. 4. Presence of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function, including but not limited to, uveitis, other macular diseases (e.g. clinically significant epiretinal membrane, full thickness macular hole or uncontrolled glaucoma/ocular hypertension). Benign conditions in the opinion of the investigator such as peripheral retina dystrophy are not exclusionary. 5. Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization. 6. History of laser therapy in the macular region. 7. Aphakia or absence of the posterior capsule. Note: Yttrium Aluminium Garnet laser posterior capsulotomy for posterior capsule opacification done at least 60 days prior to screening is not exclusionary. 8. Any ocular condition other than GA secondary to AMD that may require surgery or medical intervention during the study period or, in the opinion of the Investigator, could compromise visual function during the study period 9. Any contraindication to IVT injection including current ocular or periocular infection 10. History of prior IVT injection. 11. Prior participation in another interventional clinical study for IVT therapies in either eye (including subjects receiving sham). 12. Prior participation in another interventional clinical study for GA in either eye including investigational oral medication and placebo. 13. Participation in any systemic experimental treatment or any other systemic investigational new drug within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of study treatment. 14. Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the 24-month treatment period unlikely, or would make the subject an unsafe study candidate. 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. Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to Pegcetacoplan or any of the excipients in Pegcetacoplan solution.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of Pegcetacoplan compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by fundus autoflourescence FAF.;Secondary Objective: To evaluate the efficacy of Pegcetacoplan compared to sham injection in patients with GA secondary to AMD with respect to: • Monocular maximum reading speed (study eye), as assessed by the MNRead or Radner Reading Charts (in select countries) • Functional Reading Independence Index (FRI) score • NL-BCVA score in the study eye. To evaluate the efficacy of pegcetacoplan compared to sham injection in patients with GA secondary to AMD with respect to: • LL-BCVA in the study eye • Low luminance deficit (LLD) in the study eye • Total area of GA lesion(s) in the study eye • Monocular critical print size (study eye), as assessed by MNREAD or Radner Reading Charts (in select countries) • National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) distance activity subscale score (in select countries) To evaluate the PK of pegcetacoplan as assessed by systemic plasma concentration of pegcetacoplan (in select sites);Primary end point(s): Change from baseline to Month 12 in total area of GA lesion(s) in the study eye (in mm2) based on FAF. ;Timepoint(s) of evaluation of this end point: Month 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change from baseline in maximum monocular reading speed (study eye), as assessed by MNRead or Radner Reading Charts • Change from baseline in FRI score • Change from baseline in NL-BCVA as assessed by ETDRS chart. • Change from baseline in LL-BCVA as assessed by ETDRS chart. • Change from baseline in low luminance deficit (LLD) • Change from baseline at each planned assessment in the total area of GA lesion(s) in the study eye (in mm2) as assessed by FAF. • Change from baseline in monocular critical print size (study eye), as assessed by MNRead or Radner Reading Charts, (in selected countries). • Change from baseline in the NEI VFQ-25 distance activity subscale score (in select countries) • Systemic Plasma concentration of Pegcetacoplan;Timepoint(s) of evaluation of this end point: • Month 24 - Change from baseline in monocular reading speed MNRead or Radner Reading Charts • Month 24 - Change from baseline in FRI composite score • Month 24 - Change from baseline in NL-BCVA • Month 12, Month 24 - Change from baseline in LL-BCVA • Month 12, Month 24 - Change from baseline in LLD • Month 12, Month 24 - Change from baseline in monocular critical print size • Month 12, Month 24 - Change from baseline in NEI VFQ-25 distance activity subscale score • Over time - Systemic Plasma concentration of Pegcetacoplan. | — |
Countries
Australia, Czech Republic, France, Germany, Israel, Italy, New Zealand, Poland, Spain, United Kingdom, United States
Contacts
Public ContactClin. Development and Med. Affairs
Apellis Pharmaceuticals
Outcome results
None listed