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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)

Sponsor
Ionis Pharmaceuticals, Inc. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT03815825
Collaborator
(none)
330
Enrollment
84
Locations
2
Arms
58
Anticipated Duration (Months)
3.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

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).

Condition or Disease Intervention/Treatment Phase
Phase 2

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).

Study Design

Study Type:
Interventional
Anticipated Enrollment :
330 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
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)
Actual Study Start Date :
Mar 4, 2019
Anticipated Primary Completion Date :
Jan 1, 2024
Anticipated Study Completion Date :
Jan 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: IONIS-FB-LRx

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
IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
Other Names:
  • ISIS 696844

    		•
    Experimental: Placebo

    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
    Placebo matching solution, administered subcutaneously every 4 weeks

    Outcome Measures

    Primary Outcome Measures

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

    Secondary Outcome Measures

    1. Percentage Change from Baseline in Levels of Factor B (FB) in Plasma [Baseline and up to Week 49]

    2. Percentage Change from Baseline in Levels of Serum AH50 Activity [Baseline and up to Week 49]

    3. Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA) [Baseline and up to Week 49]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    50 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Associated Retina Consultants Ltd. Phoenix Arizona United States 85020
    2 Arizona Retina and Vitreous Consultants Phoenix Arizona United States 85021
    3 Retinal Research Institute LLC Phoenix Arizona United States 85053
    4 Barnet Dulaney Perkins Eye Center Sun City Arizona United States 85351
    5 Retina-Vitreous Associates Medical Group Beverly Hills California United States 90211
    6 Salehi Retina Institute dba Retina Associates of Southern California Huntington Beach California United States 92697
    7 UCSD - Jacobs Retina Center La Jolla California United States 92093
    8 Retina Consultants San Diego Poway California United States 92064
    9 West Coast Retina Medical Group Inc. San Francisco California United States 94109
    10 Blue Ocean Clinical Research at the Macula Center Clearwater Florida United States 33761
    11 Retina Vitreous Associates of Florida - Saint Petersburg Fort Myers Florida United States 33907
    12 Palm Beach Eye Center Lake Worth Florida United States 33461
    13 The Macula Center Lakeland Florida United States 33801
    14 Florida Eye Associates Melbourne Florida United States 32901
    15 East Florida Eye Institute Stuart Florida United States 34994
    16 USF Eye Institute Tampa Florida United States 33612
    17 Center for Retina and Macular Disease Winter Haven Florida United States 33880
    18 Southeast Retina Center, PC Augusta Georgia United States 30909-6440
    19 University Retina and Macula Associates, PC Lemont Illinois United States 60439
    20 Sabates Eye Centers Leawood Kansas United States 66211-1669
    21 Retina Specialists Baltimore Maryland United States 21204
    22 The Retina Care Center Baltimore Maryland United States 21209
    23 Cumberland Valley Retina Consultants Hagerstown Maryland United States 21740
    24 Deep Blue Retina Clinical Research Southaven Mississippi United States 38671
    25 Vision Research Center Retina Center Eye Associates of New Mexico Albuquerque New Mexico United States 87109
    26 Western Carolina Retinal Associates Asheville North Carolina United States 28803
    27 Graystone Eye Hickory North Carolina United States 28602
    28 Wake Forest Baptist Health Eye Center Winston-Salem North Carolina United States 27157
    29 University Hospitals Cleveland Medical Center Cleveland Ohio United States 44106
    30 Retina Associates of Cleveland, Inc. Cleveland Ohio United States 44130
    31 Cleveland Clinic, Cole Eye Institute Cleveland Ohio United States 44195
    32 University of Pennsylvania - Scheie Eye Institute Philadelphia Pennsylvania United States 19104
    33 UPMC Eye Center Pittsburgh Pennsylvania United States 15213
    34 Associates in Ophthalmology Ltd West Mifflin Pennsylvania United States 15122
    35 Retina Consultants of Carolina P.A. Greenville South Carolina United States 29605
    36 Charleston Neuroscience Institute Ladson South Carolina United States 29456
    37 Tennessee Retina Nashville Tennessee United States 37203
    38 Retina Research Institute of Texas Abilene Texas United States 79606
    39 Texas Retina Associates-Arlington Arlington Texas United States 76012
    40 Austin Clinical Research, LLC Austin Texas United States 78705
    41 Austin Retina Associates Austin Texas United States 78705
    42 Retina Consultants of Texas Bellaire Texas United States 77401
    43 The Retina Foundation of the Southwest Dallas Texas United States 75231
    44 Valley Retina Institute P.A. McAllen Texas United States 78503
    45 Medical Center Ophthalmology Associates San Antonio Texas United States 78240-1502
    46 Retina Center of Texas Southlake Texas United States 76051
    47 Retina Consultants of Texas The Woodlands Texas United States 77384
    48 Strategic Clinical Research Group LLC Willow Park Texas United States 76087
    49 Retina Associates of Utah Salt Lake City Utah United States 84107
    50 Piedmont Eye Center Lynchburg Virginia United States 24502
    51 Retina Institute of Virginia Richmond Virginia United States 23235
    52 Albury Eye Clinic Albury New South Wales Australia 2640
    53 Retina and Macula Specialists Hurstville New South Wales Australia 2228
    54 Marsden Eye Specialists Parramatta New South Wales Australia 2150
    55 Sydney Eye Hospital Sydney New South Wales Australia 2000
    56 Sydney Retina Clinic and Day Surgery Sydney New South Wales Australia 2000
    57 Royal Adelaide Hospital Adelaide South Australia Australia 5000
    58 Centre for Eye Research Australia, The Royal Victorian Eye & Ear Hospital East Melbourne Victoria Australia 3002
    59 Essendon Eye Clinic Essendon Victoria Australia 3040
    60 Retinology Institute Glen Iris Victoria Australia 3146
    61 Eye Surgery Associates Malvern Victoria Australia 3144
    62 Eye Surgery Associates Royal Melbourne Hospital Parkville Victoria Australia 3050
    63 Lions Eye Institute Perth Western Australia Australia 6009
    64 Kepler-University Hospital Linz Oberösterreich Austria 4020
    65 Medical University of Vienna Vienna Austria 1090
    66 Vienna Institute for Research in Ocular Surgery (VIROS) Vienna Austria 1140
    67 St Joseph's Hospital, Ivey Eye Institute London Ontario Canada N6A 4V2
    68 University of Ottawa Eye Institute Ottawa Ontario Canada K1H 8L6
    69 Retina Center of Ottawa Ottawa Ontario Canada K2B 7E9
    70 CHU de Quebec Universite Laval Québec Quebec Canada G1S 4L8
    71 Axon-Clinical s.r.o. Prague Czechia 15000
    72 Radboud Universitair Medisch Centrum Nijmegen Gelderland Netherlands 6525EX
    73 Rotterdam Eye Hospital Rotterdam Netherlands 3011 BH
    74 Southern Eye Specialists Ltd Christchurch New Zealand 8013
    75 Professor K. Gibinski University Clinical Centre Katowice Silesian Poland 40-514
    76 OFTALMIKA sp. z o.o. Bydgoszcz Poland 85-631
    77 Consorci Sanitari de Terrassa Terrassa Barcelona Spain 8227
    78 Hospital Universitario Puerta de Hierro Majadahonda Majadahonda Madrid Spain 28222
    79 Clinica Universidad De Navarra Pamplona Navarra Spain 31008
    80 Hospital IMED, Clinica Oftalvist Burjassot Valencia Spain 46100
    81 Institut de la Macula, Hospital Quiron Teknon Barcelona Spain 8022
    82 OMIQ Hospital General de Catalunya Barcelona Spain 8034
    83 Hospital Universitario Rio Hortega Valladolid Spain 47012
    84 Hospital Universitario Miguel Servet Zaragoza Spain 50009

    Sponsors and Collaborators

    • Ionis Pharmaceuticals, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Ionis Pharmaceuticals, Inc.
    ClinicalTrials.gov Identifier:
    NCT03815825
    Other Study ID Numbers:
    • ISIS 696844-CS5
    • 2020-005174-94
    First Posted:
    Jan 24, 2019
    Last Update Posted:
    Aug 22, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Macular Degeneration
    Geographic Atrophy
    Atrophy

    Study Results

    No Results Posted as of Aug 22, 2022