Geographic Atrophy, Macular Degeneration
Conditions
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).
Interventions
DRUGIONIS-FB-LRx
IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
DRUGPlacebo
Placebo matching solution, administered subcutaneously every 4 weeks
Sponsors
Ionis Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging | Week 49 |
Secondary
| Measure | Time frame |
|---|---|
| Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF) | Baseline and up to Week 57 |
| Percentage Change from Baseline in Levels of Factor B (FB) in Plasma | Baseline and up to Week 49 |
| Percentage Change from Baseline in Levels of Serum AH50 Activity | Baseline and up to Week 49 |
| Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA) | Baseline and up to Week 49 |
Countries
Australia, Austria, Canada, Czechia, Netherlands, New Zealand, Poland, Spain, United States
Outcome results
None listed