55 and older, any sex, with Geographic Atrophy or Age-related Macular Degeneration. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505Primary· Day 1 to Day 505 (starting from the day of first intravitreal injection until Day 505)
Geographic atrophy (GA) lesion growth measured by fundus autofluorescence (FAF) from baseline to Day 505.
Group
Value
95% CI
LFG316
1.95
± 1.01
Sham
1.58
± 1.12
Part A: Change From Baseline in GA Lesions Growth Measured by Fundus AutofluorescenceSecondary· Day 1 to Day 169 and Day 505 (starting from the day of first intravitreal injection until Day 505)
Mean change in GA lesion growth from baseline to Day 169 and Day 505.
Day 169 (n=68, 37)
Group
Value
95% CI
LFG316
0.99
± 0.60
Sham
0.88
± 0.77
Day 505 (n=38, 18)
Group
Value
95% CI
LFG316
2.78
± 1.28
Sham
2.03
± 1.00
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 ShamSecondary· 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: STUDY
Baseline Day 1 (n=74, 38)
Group
Value
95% CI
LFG316
43.91
± 12.93
Sham
40.26
± 14.97
Day 169 (n=71, 36)
Group
Value
95% CI
LFG316
48.38
± 11.05
Sham
42.50
± 15.14
Day 337 (n=53, 30)
Group
Value
95% CI
LFG316
47.49
± 11.25
Sham
42.97
± 14.23
Day 505 (n=40, 23)
Group
Value
95% CI
LFG316
44.73
± 11.29
Sham
43.78
± 14.11
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: FELLOWSecondary· 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
Baseline Day 1 (n=74, 38)
Group
Value
95% CI
LFG316
54.66
± 22.02
Sham
55.13
± 18.27
Day 169 (n=71, 36)
Group
Value
95% CI
LFG316
54.59
± 21.92
Sham
57.33
± 17.89
Day 337 (n=53, 30)
Group
Value
95% CI
LFG316
52.75
± 21.66
Sham
53.87
± 18.83
Day 505 (n=40, 23)
Group
Value
95% CI
LFG316
50.95
± 20.67
Sham
49.87
± 19.35
Part A: Concentrations of Total LFG316 in Blood During the Course of the StudySecondary· Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559)
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
Day 1 (n=99), h=0
Group
Value
95% CI
LFG316
0.00
± 0.00
Day 1(n= 97), h= 24
Group
Value
95% CI
LFG316
293
± 332
Day 1(n=91), h=336
Group
Value
95% CI
LFG316
560
± 238
Day 29 (n=94), h=0
Group
Value
95% CI
LFG316
289
± 214
Day 57 (n=96), h=0
Group
Value
95% CI
LFG316
382
± 218
Day 85 (n=93), h=0
Group
Value
95% CI
LFG316
436
± 210
Day 113 (n=83), h=0
Group
Value
95% CI
LFG316
433
± 219
Day 141 (n=84), h=0
Group
Value
95% CI
LFG316
451
± 218
Part A: Concentrations of Total C5 in Blood During the Course of the StudySecondary· Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559)
Summary statistic of total C5 concentrations n=number of participants, h=scheduled sampling time
Day 1 (n=99, 51), h=0
Group
Value
95% CI
LFG316
147000
± 28900
Sham
142000
± 25500
(n= 97, 51), h= 24
Group
Value
95% CI
LFG316
143000
± 31200
Sham
139000
± 27600
(n=91, 49), h=336
Group
Value
95% CI
LFG316
149000
± 41100
Sham
136000
± 25600
Day 29 (n=94, 47), h=0
Group
Value
95% CI
LFG316
145000
± 31200
Sham
142000
± 33900
Day 57 (n=96, 49), h=0
Group
Value
95% CI
LFG316
147000
± 29300
Sham
148000
± 40400
Day 85 (n=93, 46), h=0
Group
Value
95% CI
LFG316
154000
± 42500
Sham
144000
± 39100
Day 113 (n=83, 49), h=0
Group
Value
95% CI
LFG316
153000
± 46200
Sham
146000
± 42100
Day 141 (n=84, 45), h=0
Group
Value
95% CI
LFG316
144000
± 32600
Sham
141000
± 25600
Part A: Sensitivity Analysis of the Primary End Point: Mixed Effects Model for Repeated Measurements on GA Lesion Growth Measured by Fundus AutoflourescencePrimary· The primary objective was from Day 1 to Day 337, however data was captured to Day 505 as exploratory objective
Number is the Estimated Difference (95% CI) in lesion size.
Bilateral Day 169
Group
Value
95% CI
LFG316
0.975
.828 – 1.122
Sham
0.913
0.712 – 1.114
LFG316 5 Mg-Sham
0.062
-0.184 – 0.308
Bilateral Day 337
Group
Value
95% CI
LFG316
1.825
1.584 – 2.065
Sham
1.710
1.379 – 2.041
LFG316 5 Mg-Sham
0.115
-0.292 – 0.522
Bilateral Day 505
Group
Value
95% CI
LFG316
2.674
2.314 – 3.034
Sham
2.506
2.012 – 3.001
LFG316 5 Mg-Sham
0.168
-0.433 – 0.778
Overall Day 169
Group
Value
95% CI
LFG316
1.036
0.891 – 1.181
Sham
0.937
0.718 – 1.156
LFG316 5 Mg-Sham
0.099
-0.160 – 0.357
Overall Day 337
Group
Value
95% CI
LFG316
1.885
1.646 – 2.124
Sham
1.734
1.397 – 2.070
LFG316 5 Mg-Sham
0.152
-0.259 – 0.562
Overall Day 505
Group
Value
95% CI
LFG316
2.735
2.376 – 3.094
Sham
2.530
2.035 – 3.025
LFG316 5 Mg-Sham
0.205
-0.405 – 0.814
Part B: AUC (Area Under the Curve) - Summary Statistics for PK ParametersSecondary· Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
Summary statistic of total LFG316 concentrations (pharmacokinetic analysis set)
n=number of participants, h=scheduled sampling time
AUCall (hr*ng/mL)
Group
Value
95% CI
LFG316
743000
± 241000
AUClast (hr*ng/mL)
Group
Value
95% CI
LFG316
600000
± 212000
Part B: Safety and Tolerability of a Single Intravitreal (IVT) Dose of 10 mg/100 μL of LFG316 in Patients With Advanced AMD).Primary· Day 1 to Day 85
This primary outcome (for Part B) is reported under the Adverse Events section.
Total # Affected by any Serious Adverse Event
Group
Value
95% CI
Part A - LFG316 5mg
27
Sham - Part A
11
Part B - LFG316 10mg
1
Sham - Part B
0
Total # at Risk by any Serious Adverse Event
Group
Value
95% CI
Part A - LFG316 5mg
99
Sham - Part A
51
Part B - LFG316 10mg
7
Sham - Part B
1
Tmax (hr)Secondary· Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
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.
Group
Value
95% CI
LFG316
213
119 – 311
Part B: Cmax - Summary Statistic for PK ParametersSecondary· Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
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
Group
Value
95% CI
LFG316
1010
± 213
Part B: Cmax_D - Summary Statistic for PK ParametersSecondary· Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85)
Cmax\_D=ng/mL/mg
Group
Value
95% CI
LFG316
101
± 21.3
Adverse events — posted to ClinicalTrials.gov
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Part A - LFG316 5mg
Serious: 27/99 (27%)
Deaths: —
Part A - Sham
Serious: 11/51 (22%)
Deaths: —
Part B - LFG316 10mg
Serious: 1/7 (14%)
Deaths: —
Part B - Sham
Serious: 0/1 (0%)
Deaths: —
Serious adverse events (58 terms)
Reaction
System
Part A - LFG316 5mg
Part A - Sham
Part B - LFG316 10mg
Part B - Sham
Angina pectoris
Cardiac disorders
—
—
—
—
Coronary artery disease
Cardiac disorders
—
—
—
—
Abdominal pain upper
Gastrointestinal disorders
—
—
—
—
Endophthalmitis (Study Eye)
Infections and infestations
—
—
—
—
Urinary tract infection
Infections and infestations
—
—
—
—
Anaemia
Blood and lymphatic system disorders
—
—
—
—
Immune thrombocytopenic purpura
Blood and lymphatic system disorders
—
—
—
—
Atrial fibrillation
Cardiac disorders
—
—
—
—
Atrioventricular block second degree
Cardiac disorders
—
—
—
—
Cardiac failure congestive
Cardiac disorders
—
—
—
—
Cardiomyopathy
Cardiac disorders
—
—
—
—
Vertigo
Ear and labyrinth disorders
—
—
—
—
Visual acuity reduced (Fellow Eye)
Eye disorders
—
—
—
—
Visual acuity reduced (Study Eye)
Eye disorders
—
—
—
—
Constipation
Gastrointestinal disorders
—
—
—
—
Gastrointestinal haemorrhage
Gastrointestinal disorders
—
—
—
—
Gastrooesophageal reflux disease
Gastrointestinal disorders
—
—
—
—
Small intestinal obstruction
Gastrointestinal disorders
—
—
—
—
Umbilical hernia
Gastrointestinal disorders
—
—
—
—
Upper gastrointestinal haemorrhage
Gastrointestinal disorders
—
—
—
—
Vomiting
Gastrointestinal disorders
—
—
—
—
Non-cardiac chest pain
General disorders
—
—
—
—
Cellulitis
Infections and infestations
—
—
—
—
Clostridium difficile infection
Infections and infestations
—
—
—
—
Cystitis
Infections and infestations
—
—
—
—
Other adverse events (343 terms — click to expand)
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT02878616 — Study in End-stage Renal Disease Patients Awaiting Kidney Transplant to Investigate the Potential Effect of IVIG Treatme
· Phase 1
· completed
NCT02515942 — CLG561 Proof-of-Concept Study as a Monotherapy and in Combination With LFG316 in Subjects With Geographic Atrophy (GA)
· Phase 2
· completed
NCT02534909 — Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturn
· Phase 2
· completed
NCT01526889 — Safety,Tolerability and Efficacy of Intravitreal LFG316 in Patients With Active Non-infectious Intermediate-, posterior-
· Phase 2
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 5 January 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01527500.