NCT03815825

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)

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)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 332 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Macular Degeneration
• Geographic Atrophy

Interventions

Type	Name	Description
DRUG	IONIS-FB-LRx	IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
DRUG	Placebo	Placebo matching solution, administered subcutaneously every 4 weeks

Timeline

• Start date: 2019-03-04
• Primary completion: 2024-04-18
• Completion: 2024-06-12
• First posted: 2019-01-24
• Last updated: 2025-03-07

Locations

88 sites across 9 countries: United States, Australia, Austria, Canada, Czechia, Netherlands, New Zealand, Poland, Spain

Regulatory

• FDA-regulated drug study