Clinical trial NCT03446144: 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
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Geographic Atrophy
Age Related Macular Degeneration
Keywords: Geographic Atrophy
Age Related Macular Degeneration
IONIS-FB-Lrx
Complement Factor B
Lead sponsor: Ionis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2018-02-26
Enrollment: 0
Sex: ALL
Minimum age (years): 55
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Eligibility criteria: 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

Primary outcomes:
measure: Efficacy of IONIS-FB-Lrx
timeFrame: Up to 74 weeks
Secondary outcomes:
measure: Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx
timeFrame: Up to 86 weeks
measure: Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B
timeFrame: Up to 74 weeks
measure: Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD
timeFrame: Up to 74 weeks
measure: Effect of factor B reduction on other components of the complement pathways in AMD patients
timeFrame: Up to 74 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: IONIS Investigative Site
state: Arizona
city: Beverly Hills
country: United States
facility: IONIS Investigational Site
state: California
city: Encino
country: United States
facility: IONIS Investigative Site
state: California
city: Irvine
country: United States
facility: IONIS Investigative Site
state: California
city: Mountain View
country: United States
facility: IONIS Investigational Site
state: California
city: Santa Barbara
country: United States
facility: IONIS Investigational Site
state: California
city: Clearwater
country: United States
facility: IONIS Investigative Site
state: Florida
city: Tampa
country: United States
facility: IONIS Investigational Site
state: Florida
city: Augusta
country: United States
facility: IONIS Investigational Site
state: Georgia
city: Leawood
country: United States
facility: IONIS Investigational Site
state: Kansas
city: Chesterfield
country: United States
facility: IONIS Investigative Site
state: Missouri
city: New York
country: United States
facility: IONIS Investigative Site
state: New York
city: Philadelphia
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: Ladson
country: United States
facility: IONIS Investigational Site
state: South Carolina
city: Rapid City
country: United States
facility: IONIS Investigative Site
state: South Dakota
city: Arlington
country: United States
facility: IONIS Investigative Site
state: Texas
city: Austin
country: United States
facility: IONIS Investigational Site
state: Texas
city: Dallas
country: United States
facility: IONIS Investigative Site
state: Texas
city: Houston
country: United States
facility: IONIS Investigative Site
state: Texas
city: McAllen
country: United States
facility: IONIS Investigative Site
state: Texas
city: San Antonio
country: United States
facility: IONIS Investigative Site
state: Texas
city: The Woodlands
country: United States
facility: IONIS Investigative Site
state: Texas
city: Albury
country: Australia
facility: Eye Clinic Albury Wodonga
state: New South Wales
city: Liverpool
country: Australia
facility: IONIS Investigational Site
state: New South Wales
city: Paramatta
country: Australia
facility: Marsden Eye Specialists
state: New South Wales
city: Strathfield
country: Australia
facility: Strathfield Retina Clinic
state: New South Wales
city: Sydney
country: Australia
facility: Sydney Eye Hospital
state: New South Wales
city: Sydney
country: Australia
facility: Sydney Retina Clinic Day Surgery
state: New South Wales
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: East Melbourne
country: Australia
facility: The Royal Victorian Eye and Ear Hospital
state: Victoria
city: Glen Iris
country: Australia
facility: Retinology Institute
state: Victoria
city: Malvern
country: Australia
facility: Eye Surgery Associates
state: Victoria
city: Parkville
country: Australia
facility: Eye Surgery Associates
state: Victoria
city: Nedlands
country: Australia
facility: Lions Eye Institute
state: Western Australia
city: Auckland
country: New Zealand
facility: Auckland Eye
Study officials:
References: