Geographic Atrophy Secondary to Age-Related Macular Degeneration MedDRA version: 20.1 Level: LLT Classification code 10063947 Term: Geographic atrophy System Organ Class: 10015919 - Eye disorders
Conditions
Sponsors
Apellis Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Ocular-specific inclusion criteria apply to the study eye. 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. Participants 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 participants 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 participants must be: a. Women of nonchildbearing potential, or b. Women of childbearing potential (WOCBP), defined as any women who have experienced menarche and who are not permanently sterile or postmenopausal, must have 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. Inclusion criterion #2 applies to the fellow eye. 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. 1. Participants 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. Exclusion criteria 2 and 3 apply to the fellow eye.
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. Reading speed in the study eye 3. To assess the macular functional response as assessed by mesopic microperimetry in the study eye (selected participants [those who had the assessment performed in the antecedent study] only). 4. To evaluate changes in participant-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) • The rate of GA lesion growth in the study eye 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 • Monocular maximum reading speed (study eye) corrected for number of words read incorrectly as assessed by Minnesota Reading (MNRead) charts or Radner charts • Binocular maximum reading speed, corrected for number of words read incorrectly, as assessed by MNRead charts or Radner charts • The number of absolute scotomatous points (study eye) assessed by mesopic microperimetry (selected participants [those who had the assessment performed in the antecedent study] only) • Macular sensitivity (study eye) as assessed by mesopic microperimetry (selected participants [those who had the assessment performed in the antecedent study] only) • Change in additional microperimetry parameters in the study eye (eg, 95% bivariate contour ellipse area [BCEA], number of points with a clinically significant decrease in mean sensitivity) • Mean FRI Index score • NEI VFQ25 and NEI VFQ-39 (at select sites) composite score, near activity subscale score, distance activity subscale score, and NEI VFQ-25 driving subscale score ;Timepoint(s) of evaluation of this end point: •The total area of GA lesion(s) in the study eye at month 12,24,and 36 •The rate of GA lesion growth in the study eye at month 12,24,and 36 • NL-BCVA (study eye) and LL-BCVA (study eye) at month 12,24,and 36 • Monocular max reading speed (study eye) at month 12,24,and 36 • Binocular max reading speed, corrected for No of words read incorrectly at month 12,24and 36 •The number of absolute scotomatous pts (study eye) at month 12,24,and 36. • Macular sensitivity (study eye) at month 12,24,and 36 • Change in add. microperimetry parameters in the study e | — |
Countries
Argentina, Australia, Brazil, Canada, Czechia, Czech Republic, 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