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)

Age Related Macular Degeneration Clinical Trial

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)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03446144
• Other study ID #: ISIS 696844-CS3
• Status: Withdrawn
• Phase: Phase 2
• Start date: March 16, 2018
• Est. completion date: October 10, 2018

Study information

• Verified date: August 2019
• Source: Ionis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 10, 2018
• Est. primary completion date: October 10, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 55 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Age Related Macular Degeneration
• Atrophy
• Geographic Atrophy
• Macular Degeneration

Intervention

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 (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

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide	South Australia
Australia	Eye Clinic Albury Wodonga	Albury	New South Wales
Australia	The Royal Victorian Eye and Ear Hospital	East Melbourne	Victoria
Australia	Retinology Institute	Glen Iris	Victoria
Australia	IONIS Investigational Site	Liverpool	New South Wales
Australia	Eye Surgery Associates	Malvern	Victoria
Australia	Lions Eye Institute	Nedlands	Western Australia
Australia	Marsden Eye Specialists	Paramatta	New South Wales
Australia	Eye Surgery Associates	Parkville	Victoria
Australia	Strathfield Retina Clinic	Strathfield	New South Wales
Australia	Sydney Eye Hospital	Sydney	New South Wales
Australia	Sydney Retina Clinic Day Surgery	Sydney	New South Wales
New Zealand	Auckland Eye	Auckland
United States	IONIS Investigative Site	Arlington	Texas
United States	IONIS Investigational Site	Augusta	Georgia
United States	IONIS Investigational Site	Austin	Texas
United States	IONIS Investigational Site	Beverly Hills	California
United States	IONIS Investigative Site	Chesterfield	Missouri
United States	IONIS Investigative Site	Clearwater	Florida
United States	IONIS Investigative Site	Dallas	Texas
United States	IONIS Investigative Site	Encino	California
United States	IONIS Investigative Site	Houston	Texas
United States	IONIS Investigative Site	Irvine	California
United States	IONIS Investigational Site	Ladson	South Carolina
United States	IONIS Investigational Site	Leawood	Kansas
United States	IONIS Investigative Site	McAllen	Texas
United States	IONIS Investigational Site	Mountain View	California
United States	IONIS Investigative Site	New York	New York
United States	IONIS Investigative Site	Philadelphia	Pennsylvania
United States	IONIS Investigative Site	Philadelphia	Pennsylvania
United States	IONIS Investigative Site	Phoenix	Arizona
United States	IONIS Investigative Site	Pittsburgh	Pennsylvania
United States	IONIS Investigative Site	Rapid City	South Dakota
United States	IONIS Investigative Site	San Antonio	Texas
United States	IONIS Investigational Site	Santa Barbara	California
United States	IONIS Investigational Site	Tampa	Florida
United States	IONIS Investigative Site	The Woodlands	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Australia,  New Zealand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy of IONIS-FB-Lrx	The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level	Up to 74 weeks
Secondary	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	Up to 86 weeks
Secondary	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	Up to 74 weeks
Secondary	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	Up to 74 weeks
Secondary	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	Up to 74 weeks