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)

August 9, 2019 updated by: Ionis Pharmaceuticals, Inc.

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)

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

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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

Study Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Albury, New South Wales, Australia, 2640
        • Eye Clinic Albury Wodonga
      • Liverpool, New South Wales, Australia, 2170
        • Ionis Investigational Site
      • Paramatta, New South Wales, Australia, 2150
        • Marsden Eye Specialists
      • Strathfield, New South Wales, Australia, 2135
        • Strathfield Retina Clinic
      • Sydney, New South Wales, Australia, 2000
        • Sydney Eye Hospital
      • Sydney, New South Wales, Australia, 2000
        • Sydney Retina Clinic Day Surgery
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Royal Adelaide Hospital
    • Victoria
      • East Melbourne, Victoria, Australia, 3002
        • The Royal Victorian Eye and Ear Hospital
      • Glen Iris, Victoria, Australia, 3146
        • Retinology Institute
      • Malvern, Victoria, Australia, 3144
        • Eye Surgery Associates
      • Parkville, Victoria, Australia, 3050
        • Eye Surgery Associates
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Lions Eye Institute
  • New Zealand
      • Auckland, New Zealand, 1050
        • Auckland Eye
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85014-2709
        • Ionis Investigative Site
    • California
      • Beverly Hills, California, United States, 90211
        • Ionis Investigational Site
      • Encino, California, United States, 91436
        • Ionis Investigative Site
      • Irvine, California, United States, 92697
        • Ionis Investigative Site
      • Mountain View, California, United States, 94040
        • Ionis Investigational Site
      • Santa Barbara, California, United States, 93103
        • Ionis Investigational Site
    • Florida
      • Clearwater, Florida, United States, 33761
        • Ionis Investigative Site
      • Tampa, Florida, United States, 33612
        • Ionis Investigational Site
    • Georgia
      • Augusta, Georgia, United States, 30909
        • Ionis Investigational Site
    • Kansas
      • Leawood, Kansas, United States, 66211
        • Ionis Investigational Site
    • Missouri
      • Chesterfield, Missouri, United States, 63017
        • Ionis Investigative Site
    • New York
      • New York, New York, United States, 10021
        • Ionis Investigative Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Ionis Investigative Site
      • Philadelphia, Pennsylvania, United States, 19107-5109
        • Ionis Investigative Site
      • Pittsburgh, Pennsylvania, United States, 15213
        • Ionis Investigative Site
    • South Carolina
      • Ladson, South Carolina, United States, 29456
        • Ionis Investigational Site
    • South Dakota
      • Rapid City, South Dakota, United States, 57701-7374
        • Ionis Investigative Site
    • Texas
      • Arlington, Texas, United States, 76012-2505
        • Ionis Investigative Site
      • Austin, Texas, United States, 78705
        • Ionis Investigational Site
      • Dallas, Texas, United States, 75231-5078
        • Ionis Investigative Site
      • Houston, Texas, United States, 77030-2727
        • Ionis Investigative Site
      • McAllen, Texas, United States, 78503-1518
        • Ionis Investigative Site
      • San Antonio, Texas, United States, 78240-1502
        • Ionis Investigative Site
      • The Woodlands, Texas, United States, 77384-8018
        • Ionis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

55 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: TRIPLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: IONIS-FB-Lrx
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
PLACEBO_COMPARATOR: Placebo (sterile saline 0.9%)
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to 74 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to 86 weeks
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
Up to 74 weeks
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
Up to 74 weeks
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
Up to 74 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ionis Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

March 16, 2018

Primary Completion (ACTUAL)

October 10, 2018

Study Completion (ACTUAL)

October 10, 2018

Study Registration Dates

First Submitted

February 20, 2018

First Submitted That Met QC Criteria

February 20, 2018

First Posted (ACTUAL)

February 26, 2018

Study Record Updates

Last Update Posted (ACTUAL)

August 13, 2019

Last Update Submitted That Met QC Criteria

August 9, 2019

Last Verified

August 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISIS 696844-CS3

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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