An Open-Label Phase 2a Clinical Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Subjects With Primary IgA Nephropathy
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Enrollment
- 23
- Locations
- 1
- Primary Endpoint
- Percent Reduction in 24-hour Urine Protein Excretion
Study Overview
Brief Summary
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.
Detailed Description
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 Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
IONIS-FB-LRx
Intervention: IONIS-FB-LRx (Drug)
Outcomes
Primary Outcomes
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)
Secondary Outcomes
- Absolute Reduction in 24-hour Urine Protein Excretion(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 Proteinuria (UPCr Ratio)(Baseline to Week 29)
- Percent Change from Baseline in Plasma Factor B (FB)(Up to Week 29)
- Percent Change from Baseline in Plasma AH50(Up to Week 29)
- Absolute Reduction in Albuminuria (UACr Ratio)(Baseline to Week 29)