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

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 16, 2018

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 10, 2018

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 10, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 20, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 20, 2018

First Posted (ACTUAL)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 26, 2018

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 13, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 9, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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.

Clinical Trials on Geographic Atrophy

Clinical Trials on IONIS-FB-Lrx

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings