Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 and Older With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

Overview

The purpose of this study is to assess the Safety and Efficacy of IONIS-FB-Lrx for up to 120 patients with Geographic Atrophy secondary to Age Related Macular Degeneration

Full Title of Study: “A Phase 2, Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of ISIS 696844, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary (GA) to Age-Related Macular Degeneration (AMD)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: October 10, 2018

Detailed Description

This study will assess changes in complement factor B over a 69-week treatment period in a patient population 55 and older with well-demarcated Geographic Atrophy secondary to Age Related Macular Degeneration

Interventions

  • Drug: IONIS-FB-Lrx
    • Single Dose of IONIS-FB-Lrx administered once weekly for weeks 1-3, and every other week or less frequently until week 70
  • Drug: Placebo (sterline saline 0.9%)
    • Calculated volume to match active comparator. Administered subcutaneously weekly for weeks 1-3, and every other week or less frequently until week 70

Arms, Groups and Cohorts

  • Experimental: IONIS-FB-Lrx
  • Placebo Comparator: Placebo (sterile saline 0.9%)

Clinical Trial Outcome Measures

Primary Measures

  • Efficacy of IONIS-FB-Lrx
    • Time Frame: Up to 74 weeks
    • The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level

Secondary Measures

  • Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx
    • Time Frame: Up to 86 weeks
    • The safety and tolerability of IONIS-FB-Lrx will be assessed by determining the incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx
  • Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B
    • Time Frame: Up to 74 weeks
    • Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B from Baseline to Post-Treatment
  • Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD
    • Time Frame: Up to 74 weeks
    • Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD as measured by Fundus Autofluorescence from Baseline to Post-Treatment
  • Effect of factor B reduction on other components of the complement pathways in AMD patients
    • Time Frame: Up to 74 weeks
    • Effect of factor B reduction on other components of the complement pathways such as AH50, CH50 and Bb in AMD patients from Baseline to Post-Treatment

Participating in This Clinical Trial

Key Inclusion Criteria:

1. Must have given written informed consent and be able to comply with study requirements

2. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal. Males must be surgically sterile or, if engaged in sexual relations with a female of child bearing potential, the subject must be using an acceptable contraceptive method from the time of signing the informed consent form until at least a period of 13 weeks after the last dose of Study Drug (ISIS 696844 or placebo)

3. Well-demarcated Geographic Atrophy due to Age Related Macular Degeneration

Key Exclusion Criteria:

1. Clinically-significant abnormalities in medical history

2. Diagnosis of primary or secondary immunodeficiencies of B lymphocyte function, splenectomy, glomerulonephritis or history of recurrent meningococcal disease

3. Diabetes mellitus or thyroid disease unless well controlled for a period of at least 3 months

4. Clinically-significant abnormalities in screening laboratory values

5. Unwillingness to be administered, or history of a serious reaction to protocol required vaccines

6. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B

7. History or presence of a disease other than AMD in study eye

Gender Eligibility: All

Minimum Age: 55 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Ionis Pharmaceuticals, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor

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