The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) nephropathy.
This is a Phase 2, single arm open-label clinical study in up to 25 participants that will consist of a screening period, a 24-week treatment period, an optional treatment extension period of up to an additional 48 weeks, and a 12- week post-treatment follow-up evaluation period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
23
Participants will receive IONIS-FB-LRx, by subcutaneous injection (SC) at Week 1 and every 4 weeks through Week 25. Optional 48-week Extension, with drug dosing continuing every 4 weeks.
IONIS Investigative Site
Liverpool, New South Wales, Australia
IONIS Investigative Site
St Leonards, New South Wales, Australia
IONIS Investigative Site
Parkville, Victoria, Australia
IONIS Investigative Site
Vancouver, British Columbia, Canada
Percent Reduction in 24-hour Urine Protein Excretion
Time frame: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)
Absolute Reduction in 24-hour Urine Protein Excretion
Time frame: Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)
Absolute Reduction in Albuminuria (UACr Ratio)
Time frame: Baseline to Week 29
Absolute Reduction in Proteinuria (UPCr Ratio)
Time frame: Baseline to Week 29
Percent Change from Baseline in Plasma Factor B (FB)
Time frame: Up to Week 29
Percent Change from Baseline in Plasma AH50
Time frame: Up to Week 29
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IONIS Investigative Site
Toronto, Ontario, Canada
IONIS Investigative Site
Christchurch, New Zealand
IONIS Investigative Site
Singapore, Singapore