A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy

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.

Timeline

Start

2019-12-04

Primary completion

2024-02-08

Completion

2024-04-11

Outcome

Met primary endpoint

release “Phase 2 clinical study in which IONIS-FB-LRx met its primary endpoint of change in 24-hour urinary protein at 29 weeks compared to baseline.” ionis.com ↗

Publications

• Barbour SJ, Hladunewich MA, McCaleb ML, Robson R, Barrett TD, Yin L, Frazer-Abel A, Garg JP, Geary R, Schneider E, Brice GT. A single-arm phase 2 trial of an investigational RNA therapeutic to complement factor B sefaxersen for treatment of IgA nephropathy. Kidney Int. 2026 Mar;109(3):592-601. doi: 10.1016/j.kint.2025.11.017. Epub 2025 Dec 22.
• Tekendo-Ngongang C, Gleeson JG, Mignon L. Treating the Untreatable: Antisense Oligonucleotides as an Individualized Therapy for Rare Genetic Kidney Diseases. J Am Soc Nephrol. 2024 Dec 1;35(12):1774-1777. doi: 10.1681/ASN.0000000532. Epub 2024 Sep 27. No abstract available.

Drugs

Indications