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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)

Primary Purpose

Geographic Atrophy, Age Related Macular Degeneration

Status
Withdrawn
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
IONIS-FB-Lrx
Placebo (sterline saline 0.9%)
Sponsored by
Ionis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Geographic Atrophy focused on measuring Geographic Atrophy, Age Related Macular Degeneration, IONIS-FB-Lrx, Complement Factor B

Eligibility Criteria

55 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • IONIS Investigative Site
  • IONIS Investigational Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigational Site
  • IONIS Investigational Site
  • IONIS Investigative Site
  • IONIS Investigational Site
  • IONIS Investigational Site
  • IONIS Investigational Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigational Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigational Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • IONIS Investigative Site
  • Eye Clinic Albury Wodonga
  • IONIS Investigational Site
  • Marsden Eye Specialists
  • Strathfield Retina Clinic
  • Sydney Eye Hospital
  • Sydney Retina Clinic Day Surgery
  • Royal Adelaide Hospital
  • The Royal Victorian Eye and Ear Hospital
  • Retinology Institute
  • Eye Surgery Associates
  • Eye Surgery Associates
  • Lions Eye Institute
  • Auckland Eye

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

IONIS-FB-Lrx

Placebo (sterile saline 0.9%)

Arm Description

Outcomes

Primary Outcome Measures

Efficacy of IONIS-FB-Lrx
The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level

Secondary Outcome Measures

Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx
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
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
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
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

Full Information

First Posted
February 20, 2018
Last Updated
August 9, 2019
Sponsor
Ionis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03446144
Brief Title
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)
Official Title
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
Interventional

2. Study Status

Record Verification Date
August 2019
Overall Recruitment Status
Withdrawn
Why Stopped
Business objective change, no safety or efficacy concerns
Study Start Date
March 16, 2018 (Actual)
Primary Completion Date
October 10, 2018 (Actual)
Study Completion Date
October 10, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ionis Pharmaceuticals, Inc.

4. Oversight

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

5. Study Description

Brief Summary
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
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Geographic Atrophy, Age Related Macular Degeneration
Keywords
Geographic Atrophy, Age Related Macular Degeneration, IONIS-FB-Lrx, Complement Factor B

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Masking Description
Double Masked
Allocation
Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
IONIS-FB-Lrx
Arm Type
Experimental
Arm Title
Placebo (sterile saline 0.9%)
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
IONIS-FB-Lrx
Intervention Description
Single Dose of IONIS-FB-Lrx administered once weekly for weeks 1-3, and every other week or less frequently until week 70
Intervention Type
Drug
Intervention Name(s)
Placebo (sterline saline 0.9%)
Intervention Description
Calculated volume to match active comparator. Administered subcutaneously weekly for weeks 1-3, and every other week or less frequently until week 70
Primary Outcome Measure Information:
Title
Efficacy of IONIS-FB-Lrx
Description
The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level
Time Frame
Up to 74 weeks
Secondary Outcome Measure Information:
Title
Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx
Description
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
Time Frame
Up to 86 weeks
Title
Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B
Description
Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B from Baseline to Post-Treatment
Time Frame
Up to 74 weeks
Title
Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD
Description
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
Time Frame
Up to 74 weeks
Title
Effect of factor B reduction on other components of the complement pathways in AMD patients
Description
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
Time Frame
Up to 74 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Must have given written informed consent and be able to comply with study requirements 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) Well-demarcated Geographic Atrophy due to Age Related Macular Degeneration Key Exclusion Criteria: Clinically-significant abnormalities in medical history Diagnosis of primary or secondary immunodeficiencies of B lymphocyte function, splenectomy, glomerulonephritis or history of recurrent meningococcal disease Diabetes mellitus or thyroid disease unless well controlled for a period of at least 3 months Clinically-significant abnormalities in screening laboratory values Unwillingness to be administered, or history of a serious reaction to protocol required vaccines Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B History or presence of a disease other than AMD in study eye
Facility Information:
Facility Name
IONIS Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85014-2709
Country
United States
Facility Name
IONIS Investigational Site
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
IONIS Investigative Site
City
Encino
State/Province
California
ZIP/Postal Code
91436
Country
United States
Facility Name
IONIS Investigative Site
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
IONIS Investigational Site
City
Mountain View
State/Province
California
ZIP/Postal Code
94040
Country
United States
Facility Name
IONIS Investigational Site
City
Santa Barbara
State/Province
California
ZIP/Postal Code
93103
Country
United States
Facility Name
IONIS Investigative Site
City
Clearwater
State/Province
Florida
ZIP/Postal Code
33761
Country
United States
Facility Name
IONIS Investigational Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
IONIS Investigational Site
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30909
Country
United States
Facility Name
IONIS Investigational Site
City
Leawood
State/Province
Kansas
ZIP/Postal Code
66211
Country
United States
Facility Name
IONIS Investigative Site
City
Chesterfield
State/Province
Missouri
ZIP/Postal Code
63017
Country
United States
Facility Name
IONIS Investigative Site
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
IONIS Investigative Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
IONIS Investigative Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107-5109
Country
United States
Facility Name
IONIS Investigative Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
IONIS Investigational Site
City
Ladson
State/Province
South Carolina
ZIP/Postal Code
29456
Country
United States
Facility Name
IONIS Investigative Site
City
Rapid City
State/Province
South Dakota
ZIP/Postal Code
57701-7374
Country
United States
Facility Name
IONIS Investigative Site
City
Arlington
State/Province
Texas
ZIP/Postal Code
76012-2505
Country
United States
Facility Name
IONIS Investigational Site
City
Austin
State/Province
Texas
ZIP/Postal Code
78705
Country
United States
Facility Name
IONIS Investigative Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231-5078
Country
United States
Facility Name
IONIS Investigative Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030-2727
Country
United States
Facility Name
IONIS Investigative Site
City
McAllen
State/Province
Texas
ZIP/Postal Code
78503-1518
Country
United States
Facility Name
IONIS Investigative Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78240-1502
Country
United States
Facility Name
IONIS Investigative Site
City
The Woodlands
State/Province
Texas
ZIP/Postal Code
77384-8018
Country
United States
Facility Name
Eye Clinic Albury Wodonga
City
Albury
State/Province
New South Wales
ZIP/Postal Code
2640
Country
Australia
Facility Name
IONIS Investigational Site
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Marsden Eye Specialists
City
Paramatta
State/Province
New South Wales
ZIP/Postal Code
2150
Country
Australia
Facility Name
Strathfield Retina Clinic
City
Strathfield
State/Province
New South Wales
ZIP/Postal Code
2135
Country
Australia
Facility Name
Sydney Eye Hospital
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2000
Country
Australia
Facility Name
Sydney Retina Clinic Day Surgery
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2000
Country
Australia
Facility Name
Royal Adelaide Hospital
City
Adelaide
State/Province
South Australia
ZIP/Postal Code
5000
Country
Australia
Facility Name
The Royal Victorian Eye and Ear Hospital
City
East Melbourne
State/Province
Victoria
ZIP/Postal Code
3002
Country
Australia
Facility Name
Retinology Institute
City
Glen Iris
State/Province
Victoria
ZIP/Postal Code
3146
Country
Australia
Facility Name
Eye Surgery Associates
City
Malvern
State/Province
Victoria
ZIP/Postal Code
3144
Country
Australia
Facility Name
Eye Surgery Associates
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
Lions Eye Institute
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Auckland Eye
City
Auckland
ZIP/Postal Code
1050
Country
New Zealand

12. IPD Sharing Statement

Learn more about this trial

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)

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