Phase 1 Completed N=13 Treatment

Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD

Neovascular Age-Related Macular Degeneration

Source: ClinicalTrials.gov NCT02461771 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Oct 2020

Primary outcomePrimary: Number of Subjects Who Experienced Ocular and Systemic Adverse Events (AEs), Including by Severity — 2; 2; 4; 1 Participants

Summary

The objective of this study is to provide initial safety, tolerability and pharmacokinetics information of intravitreal administration of pegcetacoplan in order to support further development into larger Phase II studies for treatment of patients with AMD.

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Subjects Who Experienced Ocular and Systemic Adverse Events (AEs), Including by Severity	2; 2; 4; 1; 1; 2	—
PRIMARY Number of Dose Limiting Toxicities (DLTs)	0; 0; 0	—
PRIMARY Median Area Under the Serum Concentration-Time Curve From Time Zero to Last Measurable Concentration (AUC[0-t])	11.89; 29.95; 69.53	—
PRIMARY Median Dose Normalized AUC(0-t)	2.97; 3.00; 3.48	—
PRIMARY Maximum Observed Serum Concentration (Cmax)	0.383; 0.764; 2.140	—
PRIMARY Median Dose Normalized Cmax	0.096; 0.076; 0.107	—
PRIMARY Median Time to the Maximum Measured Serum Concentration (Tmax)	14.0; 7.9; 15.0	—
SECONDARY Median Change From Baseline in Visual Acuity for the Study Eye	1; 3; -1	—
SECONDARY Median Change From Baseline in Central Retinal Thickness, Central Retinal Lesion Thickness and Central Subfield Thickness in the Study Eye	-106; 5.0; 37.5; -79.5; 2.5; 19.0	—
SECONDARY Median Change From Baseline in Macular Cube Volume in the Study Eye	-0.4; -0.1; 0.3	—

Eligibility Criteria

Inclusion Criteria

Male or Female
Age ≥ 50 years
The presence of an active choroidal neovascular lesion secondary to AMD
On treatment with anti-VEGF therapy (Lucentis®, Eylea® or Avastin®)
Must have received at least 3 anti-VEGF treatments over the 26-week period prior to screening (Screening Visit)
Evidence that the macular fluid has responded to anti-VEGF in the past based on OCT in the opinion of PI
At screening, evidence of subretinal fluid and retinal cystic changes
Must have received anti-VEGF treatment within 10 days prior to pegcetacoplan treatment (anti-VEGF can be administered on the same day of the screening visit after the screening procedures have been completed)
OCTs of sufficient quality to allow for the assessment of the central macular fluid can be obtained
Female subjects must be:
Women of non-child-bearing potential (WONCBP), Or
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
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
Willing and able to give informed consent

Exclusion Criteria

Choroidal neovascularization associated with other ocular diseases such as pathologic myopia, ocular histoplasmosis or posterior uveitis, etc
Decreased vision due to retinal disease not attributable to choroidal neovascularization, such as nonexudative forms of AMD, geographic atrophy, inherited retinal dystrophy, uveitis or epiretinal membrane, a vitelliform-like lesion of the outer retina (e.g., as in pattern dystrophies or basal laminar drusen), idiopathic parafoveal telangiectasis, or central serous retinopathy
Additional ocular diseases that have irreversibly compromised or, during follow-up, could likely compromise the VA of the study eye including amblyopia, anterior ischemic optic neuropathy, clinically significant diabetic macular edema, severe non proliferative diabetic retinopathy, or proliferative diabetic retinopathy
Decreased vision due to significant media opacity such as corneal disease or cataract, or opacity precluding photography of the retina
Cataract surgery within three months of enrollment
Presence of any hemorrhage
History of treatment for CNV:
Previous PDT treatment within 30 days prior to enrollment in the study
Previous extrafoveal or juxtafoveal thermal laser photocoagulation within 30 days prior to enrollment in the study
Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization
Medical problems that make consistent follow-up over the treatment period unlikely (e.g. stroke, severe MI, end stage malignancy), or in general a poor medical risk because of other systemic diseases or active uncontrolled infections
Hypersensitivity to fluorescein

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02461771). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.