GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

NCT03815825 | Completed Phase 2 FDA Drug

• 2 other identifiers: ISIS 696844-CS52020-005174-94
• Study Type: interventional
• Target: 332
• Locations: 8 countries
• Sites: 77

Brief Summary

Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF).

Conditions

Macular Degeneration; Geographic Atrophy

Outcome Measures

Primary Outcomes (1)

• Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging

  Week 49

Secondary Outcomes (4)

• Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)

  Baseline and up to Week 57

• Percentage Change from Baseline in Levels of Factor B (FB) in Plasma

  Baseline and up to Week 49

• Percentage Change from Baseline in Levels of Serum AH50 Activity

  Baseline and up to Week 49

• Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)

  Baseline and up to Week 49

Study Arms (2)

IONIS-FB-LRx

EXPERIMENTAL

Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.

Drug: IONIS-FB-LRx

Placebo

EXPERIMENTAL

Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.

Drug: Placebo

Interventions

IONIS-FB-LRx DRUG

IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks

Also known as: ISIS 696844

IONIS-FB-LRx

Placebo DRUG

Placebo matching solution, administered subcutaneously every 4 weeks

Placebo

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal.
• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at least 2 weeks prior to first dose of investigational product
• Well-demarcated geographic atrophy (GA) due to AMD
• Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart
• Must have clear ocular media and adequate pupillary dilation in the study eye to permit high-quality fundus imaging

You may not qualify if:

• Clinically-significant abnormalities in medical history
• A lack of full recovery from any infection for at least 14 days prior to the Study Drug administration
• Chronic treatment with steroids, including topically or intravitreally administered
• History or presence of diabetic retinopathy or diabetic macular edema (DME)
• History or presence of a disease other than AMD that could affect vision or safety assessments
• Prior treatment with another investigational drug, biological agent, or device

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Study Sites (88)

IONIS Investigative Site

Phoenix, Arizona, 85020, United States

Location

IONIS Investigative Site

Phoenix, Arizona, 85021, United States

Location

IONIS Investigative Site

Phoenix, Arizona, 85053, United States

Location

IONIS Investigative Site

Sun City, Arizona, 85351, United States

Location

IONIS Investigative Site

Beverly Hills, California, 90211, United States

Location

IONIS Investigative Site

Huntington Beach, California, 92697, United States

Location

IONIS Investigative Site

La Jolla, California, 92093, United States

Location

IONIS Investigative Site

Long Beach, California, 90807, United States

Location

IONIS Investigative Site

Poway, California, 92064, United States

Location

IONIS Investigative Site

San Francisco, California, 94109, United States

Location

IONIS Investigative Site

Colorado Springs, Colorado, 80909, United States

Location

IONIS Investigative Site

Clearwater, Florida, 33761, United States

Location

IONIS Investigative Site

Fort Myers, Florida, 33907, United States

Location

IONIS Investigative Site

Lake Worth, Florida, 33461, United States

Location

IONIS Investigative Site

Lakeland, Florida, 33801, United States

Location

IONIS Investigative Site

Melbourne, Florida, 32901, United States

Location

IONIS Investigative Site

Stuart, Florida, 34994, United States

Location

IONIS Investigative Site

Tampa, Florida, 33612, United States

Location

IONIS Investigative Site

Winter Haven, Florida, 33880, United States

Location

IONIS Investigative Site

Augusta, Georgia, 30909, United States

Location

IONIS Investigative Site

Lemont, Illinois, 60439, United States

Location

IONIS Investigative Site

Springfield, Illinois, 62702, United States

Location

IONIS Investigative Site

Leawood, Kansas, 66211, United States

Location

IONIS Investigative Site

Baltimore, Maryland, 21204, United States

Location

IONIS Investigative Site

Baltimore, Maryland, 21209, United States

Location

IONIS Investigative Site

Hagerstown, Maryland, 21740, United States

Location

IONIS Investigative Site

Southaven, Mississippi, 38671, United States

Location

IONIS Investigative Site

Albuquerque, New Mexico, 87109, United States

Location

IONIS Investigative Site

Asheville, North Carolina, 28803, United States

Location

IONIS Investigative Site

Hickory, North Carolina, 28602, United States

Location

IONIS Investigative Site

Winston-Salem, North Carolina, 27157, United States

Location

IONIS Investigative Site

Cleveland, Ohio, 44106, United States

Location

IONIS Investigative Site

Cleveland, Ohio, 44130, United States

Location

IONIS Investigative Site

Cleveland, Ohio, 44195, United States

Location

IONIS Investigative Site

Tulsa, Oklahoma, 74114, United States

Location

IONIS Investigative Site

Philadelphia, Pennsylvania, 19104, United States

Location

IONIS Investigative Site

Pittsburgh, Pennsylvania, 15213, United States

Location

IONIS Investigative Site

West Mifflin, Pennsylvania, 15122, United States

Location

IONIS Investigative Site

Greenville, South Carolina, 29605, United States

Location

IONIS Investigative Site

Ladson, South Carolina, 29456, United States

Location

IONIS Investigative Site

Nashville, Tennessee, 37203, United States

Location

IONIS Investigative Site

Abilene, Texas, 79606, United States

Location

IONIS Investigative Site

Arlington, Texas, 76012, United States

Location

IONIS Investigative Site

Austin, Texas, 78705, United States

Location

IONIS Investigative Site

Bellaire, Texas, 77401, United States

Location

IONIS Investigative Site

Dallas, Texas, 75231, United States

Location

IONIS Investigative Site

McAllen, Texas, 78503, United States

Location

IONIS Investigative Site

San Antonio, Texas, 78240, United States

Location

IONIS Investigative Site

Southlake, Texas, 76051, United States

Location

IONIS Investigative Site

The Woodlands, Texas, 77384, United States

Location

IONIS Investigative Site

Willow Park, Texas, 76087, United States

Location

IONIS Investigative Site

Salt Lake City, Utah, 84107, United States

Location

IONIS Investigative Site

Lynchburg, Virginia, 24502, United States

Location

IONIS Investigative Site

Richmond, Virginia, 23235, United States

Location

IONIS Investigative Site

Silverdale, Washington, 98383, United States

Location

IONIS Investigative Site

Albury, New South Wales, 2640, Australia

Location

IONIS Investigative Site

Hurstville, New South Wales, 2228, Australia

Location

IONIS Investigative Site

Parramatta, New South Wales, 2150, Australia

Location

IONIS Investigative Site

Sydney, New South Wales, 2000, Australia

Location

IONIS Investigative Site

Adelaide, South Australia, 5000, Australia

Location

IONIS Investigative Site

East Melbourne, Victoria, 3002, Australia

Location

IONIS Investigative Site

Essendon, Victoria, 3040, Australia

Location

IONIS Investigative Site

Glen Iris, Victoria, 3146, Australia

Location

IONIS Investigative Site

Malvern, Victoria, 3144, Australia

Location

IONIS Investigative Site

Parkville, Victoria, 3050, Australia

Location

IONIS Investigative Site

Perth, Western Australia, 6009, Australia

Location

IONIS Investigative Site

Linz, Upper Austria, 4020, Austria

Location

IONIS Investigative Site

Klagenfurt, 9020, Austria

Location

IONIS Investigative Site

Vienna, 1090, Austria

Location

IONIS Investigative Site

Vienna, 1140, Austria

Location

IONIS Investigative Site

London, Ontario, N6A 4V2, Canada

Location

IONIS Investigative Site

Ottawa, Ontario, K1H 8L6, Canada

Location

IONIS Investigative Site

Ottawa, Ontario, K2B 7E9, Canada

Location

IONIS Investigative Site

Québec, Quebec, G1S 4L8, Canada

Location

IONIS Investigative Site

Prague, 15000, Czechia

Location

IONIS Investigative Site

Nijmegen, Gelderland, 6525EX, Netherlands

Location

IONIS Investigative Site

Rotterdam, 3011 BH, Netherlands

Location

IONIS Investigative Site

Christchurch, 8013, New Zealand

Location

IONIS Investigative Site

Katowice, Silesian, 40-514, Poland

Location

IONIS Investigative Site

Bydgoszcz, 85-631, Poland

Location

IONIS Investigative Site

Terrassa, Barcelona, 8227, Spain

Location

IONIS Investigative Site

Majadahonda, Madrid, 28222, Spain

Location

IONIS Investigative Site

Pamplona, Navarre, 31008, Spain

Location

IONIS Investigative Site

Burjassot, Valencia, 46100, Spain

Location

IONIS Investigative Site

Barcelona, 8022, Spain

Location

IONIS Investigative Site

Barcelona, 8034, Spain

Location

IONIS Investigative Site

Valladolid, 47012, Spain

Location

IONIS Investigative Site

Zaragoza, 50009, Spain

Location

Related Publications (1)

• Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

  PMID: 37314061 DERIVED

MeSH Terms

Conditions

Macular Degeneration; Geographic Atrophy

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

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

Study Record Dates

• First Submitted: January 22, 2019
• First Posted: January 24, 2019
• Study Start: March 4, 2019
• Primary Completion: April 18, 2024
• Study Completion: June 12, 2024
• Last Updated: March 7, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4); NZ (1); ES (8); US (55); CZ (1); PL (2); NL (2); CA (4)