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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
13
On treatment day, subjects will be administered a single 100 μL IVT injection of pegcetacoplan at the dose corresponding to their treatment assignment.
United States, California
Beverly Hills, California, United States
United States, Florida
Miami, Florida, United States
United States, New Hampshire
Portsmouth, New Hampshire, United States
Australia, New South Wells
Parramatta, New South Wales, Australia
Number of Subjects Who Experienced Ocular and Systemic Adverse Events (AEs), Including by Severity
Safety was assessed throughout the study. A TEAE was defined as any AE that started on/after the IVT injection of pegcetacoplan.
Time frame: Day 1 to Day 113
Number of Dose Limiting Toxicities (DLTs)
The occurrence of any of the following AEs were considered DLTs: intraocular inflammation (vitritis or uveitis), endophthalmitis, sustained elevation of intraocular pressure ≥30 millimeters (mm) of mercury, and/or sustained loss of visual acuity ≥15 letters not attributable to the injection procedure or progression of disease.
Time frame: Day 1 to Day 15
Median Area Under the Serum Concentration-Time Curve From Time Zero to Last Measurable Concentration (AUC[0-t])
The AUC(0-t) was measured using the linear trapezoidal method when concentrations were increasing and the logarithmic trapezoidal method when concentrations were decreasing. The median AUC(0-t) is presented for each cohort.
Time frame: Predose (screening), postdose Day 3 to Day 113
Median Dose Normalized AUC(0-t)
The AUC(0-t) was measured using the linear trapezoidal method when concentrations were increasing and the logarithmic trapezoidal method when concentrations were decreasing. The dose normalized AUC(0-t) was calculated for each subject by dividing the parameter by the subject's respective dose in milligrams. The median dose normalized AUC(0-t) is presented for each cohort.
Time frame: Predose (screening), postdose Day 3 to Day 113
Maximum Observed Serum Concentration (Cmax)
The median Cmax is presented for each cohort.
Time frame: Predose (screening), postdose Day 3 to Day 113
Median Dose Normalized Cmax
The dose normalized Cmax was calculated for each subject by dividing the parameter by the subject's respective dose in milligrams. The median dose normalized Cmax is presented for each cohort.
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Time frame: Predose (screening), postdose Day 3 to Day 113
Median Time to the Maximum Measured Serum Concentration (Tmax)
The median Tmax is presented for each cohort. If the maximum value occurred at more than 1 time point, Tmax was defined as the first time point with this value.
Time frame: Predose (screening), postdose Day 3 to Day 113
Median Change From Baseline in Visual Acuity for the Study Eye
Best Corrected Visual Acuity (BCVA) letter score was determined using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart assessed at a starting distance of 4 meters. The score ranges from 0 to 100 letters, lower number indicating reduced visual acuity; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.
Time frame: Day 1 to Day 113
Median Change From Baseline in Central Retinal Thickness, Central Retinal Lesion Thickness and Central Subfield Thickness in the Study Eye
Central retinal thickness, central retinal lesion thickness and central subfield thickness were determined using Spectral Domain Optical Coherence Tomography (SD-OCT).
Time frame: Day 1 to Day 113
Median Change From Baseline in Macular Cube Volume in the Study Eye
Macular cube volume was determined using SD-OCT.
Time frame: Day 1 to Day 113