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)

Ionis Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Macular Degeneration; Geographic Atrophy

Treatments: Drug: Placebo; Drug: IONIS-FB-LRx

Study type: Interventional

Funder types: Industry

Identifiers

NCT03815825

2020-005174-94 (EudraCT Number)

ISIS 696844-CS5

Details and patient eligibility

About

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).

Full 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).

Enrollment: 332 patients

Sex: All

Ages: 50+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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332 participants in 2 patient groups

IONIS-FB-LRx

Experimental group

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.

Treatment:

Drug: IONIS-FB-LRx

Placebo

Experimental group

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.

Treatment:

Drug: Placebo