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Trial record 2 of 2 for:    ionis | Geographic Atrophy
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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03815825
Recruitment Status : Active, not recruiting
First Posted : January 24, 2019
Last Update Posted : March 21, 2023
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Study Description
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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
Macular Degeneration Geographic Atrophy Drug: IONIS-FB-LRx Drug: Placebo 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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : March 4, 2019
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : July 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 Name: 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
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Primary Outcome Measures :
  1. Absolute Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging [ Time Frame: Week 49 ]

Secondary Outcome Measures :
  1. Percentage Change from Baseline in Levels of Factor B (FB) in Plasma [ Time Frame: Baseline and up to Week 49 ]
  2. Percentage Change from Baseline in Levels of Serum AH50 Activity [ Time Frame: Baseline and up to Week 49 ]
  3. Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA) [ Time Frame: Baseline and up to Week 49 ]

Eligibility Criteria
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Ages Eligible for Study:   50 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03815825


Locations
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Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03815825    
Other Study ID Numbers: ISIS 696844-CS5
2020-005174-94 ( EudraCT Number )
First Posted: January 24, 2019    Key Record Dates
Last Update Posted: March 21, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Macular Degeneration
Geographic Atrophy
Atrophy
Retinal Degeneration
 Retinal Diseases
Eye Diseases
Pathological Conditions, Anatomical