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

NCT01527500 | Completed Phase 2 Results

• 1 other identifier: CLFG316A2203
• Study Type: interventional
• Target: 158
• Locations: 1 country
• Sites: 19

Brief 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.

Conditions

Geographic Atrophy; Age-related Macular Degeneration

Keywords

AMD; Age-related Macular Degeneration; geographic atrophy; GA; Dry AMD; Blindness; Drusen; GA lesion

Outcome Measures

Primary Outcomes (3)

• 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.

  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.

  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.

  Day 1 to Day 85

Secondary Outcomes (9)

• Part A: Change From Baseline in GA Lesions Growth Measured by Fundus Autofluorescence

  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

  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, Day 169, Day 337 to Day 505

• Part A: Concentrations of Total LFG316 in Blood During the Course of the Study

  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

  Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559)

Study Arms (3)

LFG316 higher dose

EXPERIMENTAL

LFG316 10 mg/100 μL

Drug: LFG316

Sham

SHAM COMPARATOR

Sham injection

Drug: Sham

LFG316 lower dose

EXPERIMENTAL

LFG316 5 mg/ 50 μL

Drug: LFG316 Lower dose

Interventions

LFG316 DRUG

LFG316 5 mg/50 μL solution for IVT injection,

LFG316 higher dose

Sham DRUG

Sham injection (akin to intravitreal injection but without intravitreal needle; no investigational drug given)

Sham

LFG316 Lower dose DRUG

LFG316 5 mg/50 μL solution for IVT Injection

LFG316 lower dose

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (19)

Novartis Investigative Site

Phoenix, Arizona, 85014, United States

Location

Novartis Investigative Site

Phoenix, Arizona, 85020, United States

Location

Novartis Investigative Site

Tucson, Arizona, 85704-5614, United States

Location

Novartis Investigative Site

Beverly Hills, California, 90211, United States

Location

Novartis Investigative Site

Pasadena, California, 91105-3153, United States

Location

Novartis Investigative Site

Sacramento, California, 95841, United States

Location

Novartis Investigative Site

Colorado Springs, Colorado, 80909, United States

Location

Novartis Investigative Site

Fort Myers, Florida, 33912-7125, United States

Location

Novartis Investigative Site

Miami, Florida, 33143, United States

Location

Novartis Investigative Site

Winter Haven, Florida, 33880, United States

Location

Novartis Investigative Site

Atlanta, Georgia, 30342, United States

Location

Novartis Investigative Site

Leawood, Kansas, 66211, United States

Location

Novartis Investigative Site

Boston, Massachusetts, 02114, United States

Location

Novartis Investigative Site

Grand Rapids, Michigan, 49546, United States

Location

Novartis Investigative Site

Jackson, Michigan, 49202, United States

Location

Novartis Investigative Site

Charlotte, North Carolina, 28210, United States

Location

Novartis Investigative Site

Cincinnati, Ohio, 45242, United States

Location

Novartis Investigative Site

Cleveland, Ohio, 44122, United States

Location

Novartis Investigative Site

Silverdale, Washington, 98383, United States

Location

Related Publications (1)

• Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

  PMID: 37314061 DERIVED

Related Links

• A Plain Language Trial Summary is available on novartisclinicaltrials.com

MeSH Terms

Conditions

Geographic Atrophy; Macular Degeneration; Blindness

Interventions

salicylhydroxamic acid

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

Study conducted in 2 parts, Part A (n=150) \& Part B (n=8). Total participants is 158 Primary Outcome for Part B is Safety and Tolerability as presented in the Adverse events section of this report

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 2, 2012
• First Posted: February 7, 2012
• Study Start: January 25, 2012
• Primary Completion: June 24, 2015
• Study Completion: June 24, 2015
• Last Updated: January 5, 2021
• Results First Posted: July 27, 2018

Record last verified: 2019-03

Data Sharing

• IPD Sharing: Will share

Locations

US (19)