A Phase 1/2 interventional study of Pegcetacoplan in Neovascular Age-related Macular Degeneration, sponsored by Apellis Pharmaceuticals, Inc.. Terminated at 4 sites in United States. Open to participants aged 60 Years and older. Per ClinicalTrials.gov, last updated 2020-09-16.
Sponsored by Apellis Pharmaceuticals, Inc. · Phase 1/2, Interventional, and Treatment
Safety Assessment of Pegcetacoplan in Patients with Neovascular AMD
Clinical diagnosis of neovascular AMD with the following criteria met:
Female subjects must be:
Exclusion Criteria:
Drug: Pegcetacoplan
Study Drug
Also known as: APL-2
Number of Subjects Experiencing Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs) Including by Maximum Severity
TEAEs were defined as those adverse events (AEs) that developed or worsened after the first dose of study drug, up to 30 days after the last dose. The severity of the TEAEs was classified as mild (asymptomatic/mild symptoms); moderate (minimal, local/non-invasive intervention indicated); severe (medically significant but not life-threatening); life-threatening or leading to death. The number of subjects experiencing 1 TEAE in each category is presented. A maximum of 7 injections of pegcetacoplan (per subject) and a maximum of 13 injections of anti-VEGF (per subject) were received during the study.
Time frame: Day 1 up to end of study (up to 1 year).
Mean Change From Baseline in SD-OCT Central Subfield Thickness (CST) up to 12 Months
The CST was measured using SD-OCT throughout the study and the mean change from baseline at each timepoint is presented for the study eye.
Time frame: Day 1 up to Day 360 (12 months).
Number of Subjects With Clinically Significant Change From Baseline in Physical Examination Findings, Vital Signs and Laboratory Parameters
Physical examinations, vital signs and laboratory parameters were monitored throughout the study and any subjects showing a clinically significant change from baseline over the study period are presented.
Time frame: Baseline (Screening or Day 1) up to end of study (up to 1 year).
Male and female subjects aged at least 60 years with a clinical diagnosis of neovascular age-related macular degeneration in the study eye were recruited to this Phase 1b/2, open-label study at 3 study centers in the United States conducted from 14 February 2018 until the last subject last visit on 05 April 2019.
| Milestone | 15 mg Pegcetacoplan |
|---|---|
| Started | 17 |
| Completed | 0 |
| Not completed | 17 |
| Withdrew: Study/site terminated by sponsor | 14 |
| Withdrew: Withdrawal by subject | 3 |
TEAEs were defined as those adverse events (AEs) that developed or worsened after the first dose of study drug, up to 30 days after the last dose. The severity of the TEAEs was classified as mild (asymptomatic/mild symptoms); moderate (minimal, local/non-invasive intervention indicated); severe (medically significant but not life-threatening); life-threatening or leading to death. The number of subjects experiencing 1 TEAE in each category is presented. A maximum of 7 injections of pegcetacoplan (per subject) and a maximum of 13 injections of anti-VEGF (per subject) were received during the study.
| Participants | 15 mg Pegcetacoplan |
|---|---|
| Any TEAEs | 15 |
| Ocular study eye TEAE | 14 |
| Nonocular TEAE | 7 |
| TEAE at least possibly related to study drug | 5 |
| TEAE at least possibly related to injection | 8 |
| Serious TEAEs | 4 |
| TEAEs leading to study drug discontinuation | 1 |
| TEAEs leading to death | 0 |
| Maximum severity: mild | 6 |
| Maximum severity: moderate | 6 |
| Maximum severity: severe | 3 |
| Maximum severity: life-threatening | 0 |
The CST was measured using SD-OCT throughout the study and the mean change from baseline at each timepoint is presented for the study eye.
| micrometers | 15 mg Pegcetacoplan |
|---|---|
| Day 30 | 12.9 ± 39.06 |
| Day 60 | 16.1 ± 66.99 |
| Day 90 | 15.1 ± 57.44 |
| Day 120 | 31.6 ± 68.33 |
| Day 150 | 34.8 ± 90.50 |
| Day 180 | 48.3 ± 92.21 |
| Day 210 | 18.5 ± 66.06 |
| Day 240 | 34.1 ± 66.35 |
| Day 270 | 49.2 ± 58.76 |
| Day 300 | 22.2 ± 73.29 |
| Day 330 | -0.7 ± 57.17 |
| Day 360 | -10.5 ± 48.67 |
Physical examinations, vital signs and laboratory parameters were monitored throughout the study and any subjects showing a clinically significant change from baseline over the study period are presented.
| Participants | 15 mg Pegcetacoplan |
|---|---|
| Physical examination findings | 2 |
| Vital signs | 0 |
| Laboratory parameters | 0 |
Collected over Day 1 up to end of study (up to 1 year).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 15 mg Pegcetacoplan: Ocular Study Eye | 0/17 (0%) | 3/17 (17.6%) | 13/17 (76.5%) |
| 15 mg Pegcetacoplan: Ocular Fellow Eye | 0/17 (0%) | 0/17 (0%) | 3/17 (17.6%) |
| 15 mg Pecgectacoplan: Non-ocular | 0/17 (0%) | 1/17 (5.9%) | 6/17 (35.3%) |
| Event | 15 mg Pegcetacoplan: Ocular Study Eye | 15 mg Pegcetacoplan: Ocular Fellow Eye | 15 mg Pecgectacoplan: Non-ocular |
|---|---|---|---|
| UveitisEye disorders | 2/17 | 0/17 | 0/17 |
| Non-infectious endophthalmitisEye disorders | 1/17 | 0/17 | 0/17 |
| HaemoptysisRespiratory, thoracic and mediastinal disorders | 0/17 | 0/17 | 1/17 |
| Event | 15 mg Pegcetacoplan: Ocular Study Eye | 15 mg Pegcetacoplan: Ocular Fellow Eye | 15 mg Pecgectacoplan: Non-ocular |
|---|---|---|---|
| Intraocular pressure increasedInvestigations | 5/17 | 0/17 | 0/17 |
| Conjunctival haemorrhageEye disorders | 3/17 | 0/17 | 0/17 |
| UveitisEye disorders | 2/17 | 0/17 | 0/17 |
| Vision blurredEye disorders | 2/17 | 0/17 | 0/17 |
| Ocular hypertensionEye disorders | 2/17 | 0/17 | 0/17 |
| PhotopsiaEye disorders | 2/17 | 0/17 | 0/17 |
| Basal cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/17 | 0/17 | 2/17 |
| Eye painEye disorders | 1/17 | 0/17 | 0/17 |
| Posterior capsule opacificationEye disorders | 1/17 | 0/17 | 0/17 |
| Visual acuity reducedEye disorders | 1/17 | 0/17 | 0/17 |
The safety set included all subjects who received at least 1 dose of pegcetacoplan.
| Age, Continuous(years) | 15 mg Pegcetacoplan |
|---|---|
| Mean | 77.2 ± 8.76 |
| Sex: Female, Male(Participants) | 15 mg Pegcetacoplan |
|---|---|
| Female | 7 |
| Male | 10 |
| Ethnicity (NIH/OMB)(Participants) | 15 mg Pegcetacoplan |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 17 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | 15 mg Pegcetacoplan |
|---|---|
| White | 16 |
| Native Hawaiian or Other Pacific Islander | 1 |
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Apellis Pharmaceuticals, Inc.