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)

NCT03446144 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: ISIS 696844-CS3
• Study Type: interventional
• Target: N/A
• Locations: 3 countries
• Sites: 37

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

Conditions

Geographic Atrophy; Age Related Macular Degeneration

Keywords

Geographic Atrophy; Age Related Macular Degeneration; IONIS-FB-Lrx; Complement Factor B

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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

  Up to 74 weeks

• Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD

  Up to 74 weeks

• Effect of factor B reduction on other components of the complement pathways in AMD patients

  Up to 74 weeks

Study Arms (2)

IONIS-FB-Lrx

EXPERIMENTAL

Drug: IONIS-FB-Lrx

Placebo (sterile saline 0.9%)

PLACEBO COMPARATOR

Drug: Placebo (sterline saline 0.9%)

Interventions

IONIS-FB-Lrx DRUG

Single Dose of IONIS-FB-Lrx administered once weekly for weeks 1-3, and every other week or less frequently until week 70

IONIS-FB-Lrx

Placebo (sterline saline 0.9%) DRUG

Calculated volume to match active comparator. Administered subcutaneously weekly for weeks 1-3, and every other week or less frequently until week 70

Placebo (sterile saline 0.9%)

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Study Sites (37)

IONIS Investigative Site

Phoenix, Arizona, 85014-2709, United States

Location

IONIS Investigational Site

Beverly Hills, California, 90211, United States

Location

IONIS Investigative Site

Encino, California, 91436, United States

Location

IONIS Investigative Site

Irvine, California, 92697, United States

Location

IONIS Investigational Site

Mountain View, California, 94040, United States

Location

IONIS Investigational Site

Santa Barbara, California, 93103, United States

Location

IONIS Investigative Site

Clearwater, Florida, 33761, United States

Location

IONIS Investigational Site

Tampa, Florida, 33612, United States

Location

IONIS Investigational Site

Augusta, Georgia, 30909, United States

Location

IONIS Investigational Site

Leawood, Kansas, 66211, United States

Location

IONIS Investigative Site

Chesterfield, Missouri, 63017, United States

Location

IONIS Investigative Site

New York, New York, 10021, United States

Location

IONIS Investigative Site

Philadelphia, Pennsylvania, 19104, United States

Location

IONIS Investigative Site

Philadelphia, Pennsylvania, 19107-5109, United States

Location

IONIS Investigative Site

Pittsburgh, Pennsylvania, 15213, United States

Location

IONIS Investigational Site

Ladson, South Carolina, 29456, United States

Location

IONIS Investigative Site

Rapid City, South Dakota, 57701-7374, United States

Location

IONIS Investigative Site

Arlington, Texas, 76012-2505, United States

Location

IONIS Investigational Site

Austin, Texas, 78705, United States

Location

IONIS Investigative Site

Dallas, Texas, 75231-5078, United States

Location

IONIS Investigative Site

Houston, Texas, 77030-2727, United States

Location

IONIS Investigative Site

McAllen, Texas, 78503-1518, United States

Location

IONIS Investigative Site

San Antonio, Texas, 78240-1502, United States

Location

IONIS Investigative Site

The Woodlands, Texas, 77384-8018, United States

Location

Eye Clinic Albury Wodonga

Albury, New South Wales, 2640, Australia

Location

IONIS Investigational Site

Liverpool, New South Wales, 2170, Australia

Location

Marsden Eye Specialists

Paramatta, New South Wales, 2150, Australia

Location

Strathfield Retina Clinic

Strathfield, New South Wales, 2135, Australia

Location

Sydney Eye Hospital

Sydney, New South Wales, 2000, Australia

Location

Sydney Retina Clinic Day Surgery

Sydney, New South Wales, 2000, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

The Royal Victorian Eye and Ear Hospital

East Melbourne, Victoria, 3002, Australia

Location

Retinology Institute

Glen Iris, Victoria, 3146, Australia

Location

Eye Surgery Associates

Malvern, Victoria, 3144, Australia

Location

Eye Surgery Associates

Parkville, Victoria, 3050, Australia

Location

Lions Eye Institute

Nedlands, Western Australia, 6009, Australia

Location

Auckland Eye

Auckland, 1050, New Zealand

Location

MeSH Terms

Conditions

Geographic Atrophy; Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double Masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2018
• First Posted: February 26, 2018
• Study Start: March 16, 2018
• Primary Completion: October 10, 2018
• Study Completion: October 10, 2018
• Last Updated: August 13, 2019

Record last verified: 2019-08

Locations

AU (12); US (24); NZ (1)