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

Phase 2

Completed

• Conditions: Primary IgA Nephropathy
• Interventions: Drug: IONIS-FB-LRx

• Registration Number: NCT04014335
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

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 Timeline

• Study First Submitted: 2019-07-08
• Study First Submitted QC: 2019-07-08
• Study First Posted: 2019-07-10
• Study Start: 2019-12-04
• Primary Completion: 2024-02-08
• Study Completion: 2024-04-11
• Disposition First Posted: 2024-05-14
• Status Verified: 2025-01
• Disposition First Submitted: 2025-01-17
• Last Update Submitted: 2025-01-17
• Last Update Posted: 2025-01-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
IONIS-FB-LRx	IONIS-FB-LRx	-

Primary Outcome Measures

Name	Time	Method
Percent 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)

Secondary Outcome Measures

Name	Time	Method
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)
Percent Change from Baseline in Plasma Factor B (FB)	Up to Week 29
Absolute Reduction in Albuminuria (UACr Ratio)	Baseline to Week 29
Absolute Reduction in Proteinuria (UPCr Ratio)	Baseline to Week 29
Percent Change from Baseline in Plasma AH50	Up to Week 29

Trial Locations

Locations (1)

IONIS Investigative Site; 🇸🇬 Singapore, Singapore