This study was conducted in two parts; Part A and Part B: Part B was initially planned to include two cohorts. Cohort 2 was cancelled following an interim analysis for efficacy in Part A of the study, and not due to any safety issues or concerns. Cohort 2 is not referred to again and part B cohort 1 is referred to as part B alone in the remainder of the document and is the subject of this report. Part B was conducted to assess the safety and tolerability of a single intravitreal (IVT) LFG316 10 mg/100 µL injection. There was no efficacy evaluation in Part B. The study employed a multicenter, randomized, sham - controlled, single masked design. Eight patients with advanced AMD were planned to be randomized in a 3:1 ratio to receive a single IVT dose of LFG316 (10 mg/100 µL) or sham injection. Patients assigned to a sham injection were treated the same as those assigned to LFG316, except that the hub of an empty syringe (without needle) was placed against the eye instead of the IVT injection.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
158
Novartis Investigative Site
Phoenix, Arizona, United States
Novartis Investigative Site
Phoenix, Arizona, United States
Novartis Investigative Site
Tucson, Arizona, United States
Novartis Investigative Site
Beverly Hills, California, United States
Novartis Investigative Site
Pasadena, California, United States
Novartis Investigative Site
Sacramento, California, United States
Novartis Investigative Site
Colorado Springs, Colorado, United States
Novartis Investigative Site
Fort Myers, Florida, United States
Novartis Investigative Site
Miami, Florida, United States
Novartis Investigative Site
Winter Haven, Florida, United States
...and 9 more locations
Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505
Geographic atrophy (GA) lesion growth measured by fundus autofluorescence (FAF) from baseline to Day 505.
Time frame: Day 1 to Day 505 (starting from the day of first intravitreal injection until Day 505)
Part A: Sensitivity Analysis of the Primary End Point: Mixed Effects Model for Repeated Measurements on GA Lesion Growth Measured by Fundus Autoflourescence
Number is the Estimated Difference (95% CI) in lesion size.
Time frame: The primary objective was from Day 1 to Day 337, however data was captured to Day 505 as exploratory objective
Part B: Safety and Tolerability of a Single Intravitreal (IVT) Dose of 10 mg/100 μL of LFG316 in Patients With Advanced AMD).
This primary outcome (for Part B) is reported under the Adverse Events section.
Time frame: Day 1 to Day 85
Part A: Change From Baseline in GA Lesions Growth Measured by Fundus Autofluorescence
Mean change in GA lesion growth from baseline to Day 169 and Day 505.
Time frame: Day 1 to Day 169 and Day 505 (starting from the day of first intravitreal injection until Day 505)
Part A: Change in Best Corrected Visual Acuity (BCVA) as Measured by the EDTRS (Early Treatment of Diabetic Retinopathy Study) Scale From Baseline to Days 169, 337 & 505 in Patients Receiving Every 28 Days, Successive IVT Doses of LFG316 Compared to Sham
Part A: Summary of best corrected visual acuity over time, statistical analysis of change in best corrected visual acuity over time Parameter: Visual Acuity (EDTRS letter) BCVA scale is 0-100, worst is 0 and best 100 Eye: STUDY
Time frame: Baseline Day 1, Day 169, Day 337 to Day 505
Part A: Summary of Best Corrected Visual Acuity Over Time, Statistical Analysis of Change in Best Corrected Visual Acuity Over Time Parameter: Visual Acuity (EDTRS Letter) BCVA Scale is 0-100, Worst is 0 and Best 100 Eye: FELLOW
Part A: Summary of best corrected visual acuity over time, statistical analysis of change in best corrected visual acuity over time Parameter: Visual Acuity (EDTRS letter) BCVA scale is 0-100, worst is 0 and best 100 Eye: FELLOW
Time frame: Baseline Day 1, Day 169, Day 337 to Day 505
Part A: Concentrations of Total LFG316 in Blood During the Course of the Study
Summary statistic of total LFG316 concentrations (pharmacokinetic analysis set) n=number of participants, h=hours after the last administered dose e.g.; 0.0 means just before dosing. If the mean concentration is 0.00, that means there is no drug in the bloodstream
Time frame: Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559)
Part A: Concentrations of Total C5 in Blood During the Course of the Study
Summary statistic of total C5 concentrations n=number of participants, h=scheduled sampling time
Time frame: Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559)
Part B: AUC (Area Under the Curve) - Summary Statistics for PK Parameters
Summary statistic of total LFG316 concentrations (pharmacokinetic analysis set) n=number of participants, h=scheduled sampling time
Time frame: Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
Tmax (hr)
PART B: Tmax (Time of Maximum concentration observed) This is the highest concentration of drug in the blood that is measured after a dose. Cmax usually happens within a few hours after the dose is taken. The time that Cmax happens is referred to as Tmax. For some antiretroviral drugs, a high Cmax is thought to increase the risk of side effects from the drug.
Time frame: Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
Part B: Cmax - Summary Statistic for PK Parameters
Summary statistic for Part B of total LFG316 concentrations (pharmacokinetic analysis set) Cmax is the maximum (or peak) serum concentration that a drug achieves in a specified compartment or test area of the body after the drug has been administrated and before the administration of a second dose
Time frame: Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
Part B: Cmax_D - Summary Statistic for PK Parameters
Cmax\_D=ng/mL/mg
Time frame: Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
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