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: Ocular-specific inclusion criteria apply to the study eye only. 1. Participated in APL2-103 (NCT03777332) or completed the treatment at Month 24 of either APL2-303 (Derby, NCT03525613) or APL2-304 (Oaks, NCT03525600). Specifically, for the APL2-303 and APL2-304 studies, the following criterion also applies: a. Subjects who did not permanently discontinue treatment but missed the Month 24 visit are also eligible to participate in this extension study; however, to be eligible, these subjects must be screened within 60 days from the last day of the expected Month 24 visit window for the antecedent study. 2. Clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the investigator. 3. Female subjects must be: a. Women of nonchildbearing potential, or b. Women of childbearing potential (WOCBP) with a negative serum pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study and 90 days after their last dose of pegcetacoplan, and refrain from breastfeeding for the duration of the study. 4. Males with female partners of childbearing potential must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and for 90 days after their last dose of pegcetacoplan. 5. 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 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1160
Exclusion criteria
Exclusion criteria: Ocular-specific exclusion criteria apply to the study eye only. 1. Subjects who permanently discontinued the study drug prior to Month 24 in the APL2-304 or APL2-304 studies and only for safety assessments. Temporary pause of the study drug is not exclusionary. 2. Presence of an active ocular disease that, in the opinion of the investigator, compromises or confounds visual function, including, but not limited to, macular hole or other macular diseases (eg, clinically significant epiretinal membrane). Benign conditions in the opinion of the investigator such as peripheral retinal dystrophy are not exclusionary. 3. Any contraindication to IVT injection including current ocular or periocular infection. 4. Medical or psychiatric condition that, in the opinion of the investigator, is clinically significant and not suitable for study participation or make consistent follow-up over the 36-month treatment period unlikely. 5. Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to pegcetacoplan or any of the excipients in pegcetacoplan solution. 6. Pregnancy, breastfeeding, or positive pregnancy test.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety of IVT injected pegcetacoplan.;Secondary Objective: 1. To assess changes in the total area of geographic atrophy (GA) lesion in the study eye measured by fundus autofluorescence (FAF). 2. To assess changes in visual function as measured by: a. Normal-luminance best-corrected visual acuity score (NL-BCVA) in the study eye b. Low-luminance best-corrected visual acuity score (LL-BCVA) in the study eye c. Low-luminance deficit (LLD) in the study eye d. Reading speed in the study eye 3. To assess the macular functional response as assessed by mesopic microperimetry in the study eye (selected subjects [those who had the assessment performed in the antecedent study] only). 4. To evaluate changes in subject-reported outcomes as measured by: a. National Eye Institute Visual Functioning Questionnaire 25 Item Version (NEI VFQ-25) b. Functional Reading Independence (FRI) Index.;Primary end point(s): Incidence and severity of ocular and systemic AEs;Timepoint(s) of evaluation of this end point: Incidence and severity of ocular and systemic AEs (time frame: up to 36 months) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • The total area of GA lesion(s) in the study eye (in mm2) as assessed by fundus autofluorescence (FAF) • NL-BCVA score (study eye) as assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) chart • LL-BCVA score (study eye) as assessed by ETDRS chart • LLD (study eye) • Monocular maximum reading speed (study eye) and monocular critical print size, as assessed by Minnesota Reading (MNRead) charts or Radner • Binocular maximum reading speed and binocular critical print size, as assessed by MNRead charts or Radner. • The number of absolute scotomatous points (study eye) assessed by mesopic microperimetry (selected subjects [those who had the assessment performed in the antecedent study] only) • Macular sensitivity (study eye) as assessed by mesopic microperimetry (selected subjects [those who had the assessment performed in the antecedent study] only) • Mean FRI Index score • NEI VFQ-25 composite score, near activity subscale score, and distance activity subscale score;Timepoint(s) of evaluation of this end point: •The total area of GA lesion(s) in the study eye (in mm2) as assessed by FAF at Month 12, 24 and 36. • NL-BCVA score (study eye) and LL-BCVA score (study eye) as assessed by ETDRS chart at Month 12, 24 and 36. • LLD (study eye) at Month 12, 24 and 36. • Monocular maximum reading speed (study eye) and monocular critical print size at Month 12, 24 and 36. • Binocular maximum reading speed and binocular critical print size at Month 12, 24 and 36. The number of absolute scotomatous points (study eye) at Month 12, 24, and 36. • Macular sensitivity (study eye) at Month 12, 24, and 36. • Mean FRI Index score at Month 12, 24, and 36 • NEI VFQ-25 composite score, near activity subscale score, and distance activity subscale score at Month 12, 24, and 36. | — |
Countries
Argentina, Australia, Brazil, Canada, Czechia, France, Germany, Israel, Italy, Netherlands, New Zealand, Poland, Spain, United Kingdom, United States
Contacts
Public ContactClin. Development and Med. Affairs
Apellis Pharmaceuticals
Outcome results
None listed