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Phase 2 N=150 Randomized Single-blind Treatment

Intravitreal LFG316 in Patients With Age-related Macular Degeneration (AMD)

Geographic Atrophy · Age-related Macular Degeneration

Enrolled (actual)
150
Serious AEs
24.7%
Results posted
Jul 2018
Primary outcome: Primary: Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505 — 1.95; 1.58 mm^2

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
LFG316 (Drug); Sham (Drug); LFG316 Lower dose (Drug)
Age
Adult, Older Adult · 55+ yrs
Sex
All
Sponsor
Novartis Pharmaceuticals
Primary completion
Jun 2015

Outcome Measures

OutcomeResultp-value
PRIMARY
Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505
1.95; 1.58
PRIMARY
Part A: Sensitivity Analysis of the Primary End Point: Mixed Effects Model for Repeated Measurements on GA Lesion Growth Measured by Fundus Autoflourescence
0.975; 0.913; 0.062; 1.825; 1.710; 0.115
PRIMARY
Part B: Safety and Tolerability of a Single Intravitreal (IVT) Dose of 10 mg/100 μL of LFG316 in Patients With Advanced AMD).
27; 11; 1; 0; 99; 51
SECONDARY
Part A: Change From Baseline in GA Lesions Growth Measured by Fundus Autofluorescence
0.99; 0.88; 2.78; 2.03
SECONDARY
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
43.91; 40.26; 48.38; 42.50; 47.49; 42.97
SECONDARY
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
54.66; 55.13; 54.59; 57.33; 52.75; 53.87
SECONDARY
Part A: Concentrations of Total LFG316 in Blood During the Course of the Study
0.00; 293; 560; 289; 382; 436
SECONDARY
Part A: Concentrations of Total C5 in Blood During the Course of the Study
147000; 142000; 143000; 139000; 149000; 136000
SECONDARY
Part B: AUC (Area Under the Curve) - Summary Statistics for PK Parameters
743000; 600000
SECONDARY
Tmax (hr)
213
SECONDARY
Part B: Cmax - Summary Statistic for PK Parameters
1010
SECONDARY
Part B: Cmax_D - Summary Statistic for PK Parameters
101

Summary

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.

Eligibility Criteria

Inclusion Criteria

  • Diagnosis of AMD if enrolled in Part B of study
  • Geographic atrophy in at least one eye if enrolled in Part A of study
  • ETDRS best corrected visual acuity of 60 letters or worse (~≤ 20/63)

Exclusion Criteria

  • Retinal disease other than AMD
  • History of choroidal neovascularization
  • Severe cataract
  • History of infectious uveitis or endophthalmitis
  • Eye surgery in the non-study eye within 30 days prior to study
  • Eye surgery or IVT injection in the study eye within 90 days prior to study
  • Other protocol-defined inclusion/exclusion criteria may apply
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01527500). 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.

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