A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

Overview

The purpose of the study is to evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF).

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Double (Participant, Investigator)
  • Study Primary Completion Date: October 2022

Detailed Description

The study will assess the rate of change of the area of GA secondary to AMD by measuring FAF in up to 330 participants with GA due to AMD being treated with IONIS-FB-LRx. The study is a Phase 2, double-masked, randomized, stratified, placebo-controlled study conducted at multiple centers. It is an adaptive design in which three dose levels will be evaluated in a subset of patients (Stage 1) and, following an interim analysis, the number of patients in two of the dose cohorts will be expanded (Stage 2).

Interventions

  • Drug: IONIS-FB-LRx
    • IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
  • Drug: Placebo
    • Placebo matching solution, administered subcutaneously every 4 weeks

Arms, Groups and Cohorts

  • Experimental: IONIS-FB-LRx
    • Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
  • Experimental: Placebo
    • Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.

Clinical Trial Outcome Measures

Primary Measures

  • Absolute Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
    • Time Frame: Week 49

Secondary Measures

  • Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
    • Time Frame: Baseline and up to Week 49
  • Percentage Change from Baseline in Levels of Serum AH50 Activity
    • Time Frame: Baseline and up to Week 49
  • Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
    • Time Frame: Baseline and up to Week 49

Participating in This Clinical Trial

Inclusion Criteria

  • Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal.
  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at least 2 weeks prior to first dose of investigational product
  • Well-demarcated geographic atrophy (GA) due to AMD
  • Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart
  • Must have clear ocular media and adequate pupillary dilation in the study eye to permit high-quality fundus imaging

Exclusion Criteria

  • Clinically-significant abnormalities in medical history
  • A lack of full recovery from any infection for at least 14 days prior to the Study Drug administration
  • Chronic treatment with steroids, including topically or intravitreally administered
  • History or presence of diabetic retinopathy or diabetic macular edema (DME)
  • History or presence of a disease other than AMD that could affect vision or safety assessments
  • Prior treatment with another investigational drug, biological agent, or device
  • Other protocol-specified inclusion/exclusion criteria may apply

Gender Eligibility: All

Minimum Age: 50 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
  • Overall Contact(s)
    • Ionis Pharmaceuticals, 800-679-4747, patients@ionisph.com

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