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)

Phase 2

Withdrawn

• Conditions: Geographic Atrophy; Age Related Macular Degeneration
• Interventions: Drug: IONIS-FB-Lrx; Drug: Placebo (sterline saline 0.9%)

• Registration Number: NCT03446144
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

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

Study Timeline

• Study First Submitted: 2018-02-20
• Study First Submitted QC: 2018-02-20
• Study First Posted: 2018-02-26
• Study Start: 2018-03-16
• Primary Completion: 2018-10-10
• Study Completion: 2018-10-10
• Status Verified: 2019-08
• Last Update Submitted: 2019-08-09
• Last Update Posted: 2019-08-13

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
IONIS-FB-Lrx	IONIS-FB-Lrx	-
Placebo (sterile saline 0.9%)	Placebo (sterline saline 0.9%)	-

Primary Outcome Measures

Name	Time	Method
Efficacy of IONIS-FB-Lrx	Up to 74 weeks	The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level

Secondary Outcome Measures

Name	Time	Method
Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx	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
Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B	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
Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD	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
Effect of factor B reduction on other components of the complement pathways in AMD patients	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

Trial Locations

Locations (13)

IONIS Investigative Site; 🇺🇸 The Woodlands, Texas, United States

IONIS Investigational Site; 🇦🇺 Liverpool, New South Wales, Australia

Eye Clinic Albury Wodonga; 🇦🇺 Albury, New South Wales, Australia

Marsden Eye Specialists; 🇦🇺 Paramatta, New South Wales, 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, Australia

Royal Adelaide Hospital; 🇦🇺 Adelaide, South Australia, Australia

The Royal Victorian Eye and Ear Hospital; 🇦🇺 East Melbourne, Victoria, Australia

Retinology Institute; 🇦🇺 Glen Iris, Victoria, Australia

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