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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)

Primary Purpose

Macular Degeneration, Geographic Atrophy

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
IONIS-FB-LRx
Placebo
Sponsored by
Ionis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Macular Degeneration

Eligibility Criteria

50 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • Other protocol-specified inclusion/exclusion criteria may apply

Sites / Locations

  • IONIS Investigative Site
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Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

IONIS-FB-LRx

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
Percentage Change from Baseline in Levels of Serum AH50 Activity
Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)

Full Information

First Posted
January 22, 2019
Last Updated
August 3, 2023
Sponsor
Ionis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03815825
Brief Title
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)
Official Title
A Phase 2, Randomized Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
March 4, 2019 (Actual)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
July 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ionis Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).
Detailed Description
The study will assess the rate of change of the area of GA secondary to AMD by measuring FAF in up to 330 participants with GA due to AMD being treated with IONIS-FB-LRx. The study is a Phase 2, double-masked, randomized, stratified, placebo-controlled study conducted at multiple centers. It is an adaptive design in which three dose levels will be evaluated in a subset of participants (Stage 1) and, following an interim analysis, the number of participants in two of the dose cohorts will be expanded (Stage 2).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Macular Degeneration, Geographic Atrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
332 (Actual)

8. Arms, Groups, and Interventions

Arm Title
IONIS-FB-LRx
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
IONIS-FB-LRx
Other Intervention Name(s)
ISIS 696844
Intervention Description
IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matching solution, administered subcutaneously every 4 weeks
Primary Outcome Measure Information:
Title
Absolute Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
Time Frame
Week 49
Secondary Outcome Measure Information:
Title
Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
Time Frame
Baseline and up to Week 49
Title
Percentage Change from Baseline in Levels of Serum AH50 Activity
Time Frame
Baseline and up to Week 49
Title
Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
Time Frame
Baseline and up to Week 49

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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 Other protocol-specified inclusion/exclusion criteria may apply
Facility Information:
Facility Name
IONIS Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85020
Country
United States
Facility Name
IONIS Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85021
Country
United States
Facility Name
IONIS Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85053
Country
United States
Facility Name
IONIS Investigative Site
City
Sun City
State/Province
Arizona
ZIP/Postal Code
85351
Country
United States
Facility Name
IONIS Investigative Site
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
IONIS Investigative Site
City
Huntington Beach
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
IONIS Investigative Site
City
La Jolla
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
IONIS Investigative Site
City
Long Beach
State/Province
California
ZIP/Postal Code
90807
Country
United States
Facility Name
IONIS Investigative Site
City
Poway
State/Province
California
ZIP/Postal Code
92064
Country
United States
Facility Name
IONIS Investigative Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94109
Country
United States
Facility Name
IONIS Investigative Site
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80909
Country
United States
Facility Name
IONIS Investigative Site
City
Clearwater
State/Province
Florida
ZIP/Postal Code
33761
Country
United States
Facility Name
IONIS Investigative Site
City
Fort Myers
State/Province
Florida
ZIP/Postal Code
33907
Country
United States
Facility Name
IONIS Investigative Site
City
Lake Worth
State/Province
Florida
ZIP/Postal Code
33461
Country
United States
Facility Name
IONIS Investigative Site
City
Lakeland
State/Province
Florida
ZIP/Postal Code
33801
Country
United States
Facility Name
IONIS Investigative Site
City
Melbourne
State/Province
Florida
ZIP/Postal Code
32901
Country
United States
Facility Name
IONIS Investigative Site
City
Stuart
State/Province
Florida
ZIP/Postal Code
34994
Country
United States
Facility Name
IONIS Investigative Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
IONIS Investigative Site
City
Winter Haven
State/Province
Florida
ZIP/Postal Code
33880
Country
United States
Facility Name
IONIS Investigative Site
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30909-6440
Country
United States
Facility Name
IONIS Investigative Site
City
Lemont
State/Province
Illinois
ZIP/Postal Code
60439
Country
United States
Facility Name
IONIS Investigative Site
City
Springfield
State/Province
Illinois
ZIP/Postal Code
62702
Country
United States
Facility Name
IONIS Investigative Site
City
Leawood
State/Province
Kansas
ZIP/Postal Code
66211-1669
Country
United States
Facility Name
IONIS Investigative Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21204
Country
United States
Facility Name
IONIS Investigative Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21209
Country
United States
Facility Name
IONIS Investigative Site
City
Hagerstown
State/Province
Maryland
ZIP/Postal Code
21740
Country
United States
Facility Name
IONIS Investigative Site
City
Southaven
State/Province
Mississippi
ZIP/Postal Code
38671
Country
United States
Facility Name
IONIS Investigative Site
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87109
Country
United States
Facility Name
IONIS Investigative Site
City
Asheville
State/Province
North Carolina
ZIP/Postal Code
28803
Country
United States
Facility Name
IONIS Investigative Site
City
Hickory
State/Province
North Carolina
ZIP/Postal Code
28602
Country
United States
Facility Name
IONIS Investigative Site
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
IONIS Investigative Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
IONIS Investigative Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44130
Country
United States
Facility Name
IONIS Investigative Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
IONIS Investigative Site
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74114
Country
United States
Facility Name
IONIS Investigative Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
IONIS Investigative Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
IONIS Investigative Site
City
West Mifflin
State/Province
Pennsylvania
ZIP/Postal Code
15122
Country
United States
Facility Name
IONIS Investigative Site
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29605
Country
United States
Facility Name
IONIS Investigative Site
City
Ladson
State/Province
South Carolina
ZIP/Postal Code
29456
Country
United States
Facility Name
IONIS Investigative Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Facility Name
IONIS Investigative Site
City
Abilene
State/Province
Texas
ZIP/Postal Code
79606
Country
United States
Facility Name
IONIS Investigative Site
City
Arlington
State/Province
Texas
ZIP/Postal Code
76012
Country
United States
Facility Name
IONIS Investigative Site
City
Austin
State/Province
Texas
ZIP/Postal Code
78705
Country
United States
Facility Name
IONIS Investigative Site
City
Bellaire
State/Province
Texas
ZIP/Postal Code
77401
Country
United States
Facility Name
IONIS Investigative Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
IONIS Investigative Site
City
McAllen
State/Province
Texas
ZIP/Postal Code
78503
Country
United States
Facility Name
IONIS Investigative Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78240-1502
Country
United States
Facility Name
IONIS Investigative Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78240
Country
United States
Facility Name
IONIS Investigative Site
City
Southlake
State/Province
Texas
ZIP/Postal Code
76051
Country
United States
Facility Name
IONIS Investigative Site
City
The Woodlands
State/Province
Texas
ZIP/Postal Code
77384
Country
United States
Facility Name
IONIS Investigative Site
City
Willow Park
State/Province
Texas
ZIP/Postal Code
76087
Country
United States
Facility Name
IONIS Investigative Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Facility Name
IONIS Investigative Site
City
Lynchburg
State/Province
Virginia
ZIP/Postal Code
24502
Country
United States
Facility Name
IONIS Investigative Site
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23235
Country
United States
Facility Name
IONIS Investigative Site
City
Silverdale
State/Province
Washington
ZIP/Postal Code
98383
Country
United States
Facility Name
IONIS Investigative Site
City
Albury
State/Province
New South Wales
ZIP/Postal Code
2640
Country
Australia
Facility Name
IONIS Investigative Site
City
Hurstville
State/Province
New South Wales
ZIP/Postal Code
2228
Country
Australia
Facility Name
IONIS Investigative Site
City
Parramatta
State/Province
New South Wales
ZIP/Postal Code
2150
Country
Australia
Facility Name
IONIS Investigative Site
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2000
Country
Australia
Facility Name
IONIS Investigative Site
City
Adelaide
State/Province
South Australia
ZIP/Postal Code
5000
Country
Australia
Facility Name
IONIS Investigative Site
City
East Melbourne
State/Province
Victoria
ZIP/Postal Code
3002
Country
Australia
Facility Name
IONIS Investigative Site
City
Essendon
State/Province
Victoria
ZIP/Postal Code
3040
Country
Australia
Facility Name
IONIS Investigative Site
City
Glen Iris
State/Province
Victoria
ZIP/Postal Code
3146
Country
Australia
Facility Name
IONIS Investigative Site
City
Malvern
State/Province
Victoria
ZIP/Postal Code
3144
Country
Australia
Facility Name
IONIS Investigative Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
IONIS Investigative Site
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
IONIS Investigative Site
City
Linz
State/Province
Oberösterreich
ZIP/Postal Code
4020
Country
Austria
Facility Name
IONIS Investigative Site
City
Klagenfurt
ZIP/Postal Code
9020
Country
Austria
Facility Name
IONIS Investigative Site
City
Vienna
ZIP/Postal Code
1090
Country
Austria
Facility Name
IONIS Investigative Site
City
Vienna
ZIP/Postal Code
1140
Country
Austria
Facility Name
IONIS Investigative Site
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 4V2
Country
Canada
Facility Name
IONIS Investigative Site
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L6
Country
Canada
Facility Name
IONIS Investigative Site
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K2B 7E9
Country
Canada
Facility Name
IONIS Investigative Site
City
Québec
State/Province
Quebec
ZIP/Postal Code
G1S 4L8
Country
Canada
Facility Name
IONIS Investigative Site
City
Prague
ZIP/Postal Code
15000
Country
Czechia
Facility Name
IONIS Investigative Site
City
Nijmegen
State/Province
Gelderland
ZIP/Postal Code
6525EX
Country
Netherlands
Facility Name
IONIS Investigative Site
City
Rotterdam
ZIP/Postal Code
3011 BH
Country
Netherlands
Facility Name
IONIS Investigative Site
City
Christchurch
ZIP/Postal Code
8013
Country
New Zealand
Facility Name
IONIS Investigative Site
City
Katowice
State/Province
Silesian
ZIP/Postal Code
40-514
Country
Poland
Facility Name
IONIS Investigative Site
City
Bydgoszcz
ZIP/Postal Code
85-631
Country
Poland
Facility Name
IONIS Investigative Site
City
Terrassa
State/Province
Barcelona
ZIP/Postal Code
8227
Country
Spain
Facility Name
IONIS Investigative Site
City
Majadahonda
State/Province
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
IONIS Investigative Site
City
Pamplona
State/Province
Navarra
ZIP/Postal Code
31008
Country
Spain
Facility Name
IONIS Investigative Site
City
Burjassot
State/Province
Valencia
ZIP/Postal Code
46100
Country
Spain
Facility Name
IONIS Investigative Site
City
Barcelona
ZIP/Postal Code
8022
Country
Spain
Facility Name
IONIS Investigative Site
City
Barcelona
ZIP/Postal Code
8034
Country
Spain
Facility Name
IONIS Investigative Site
City
Valladolid
ZIP/Postal Code
47012
Country
Spain
Facility Name
IONIS Investigative Site
City
Zaragoza
ZIP/Postal Code
50009
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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)

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