IONIS-FB-LRx for IgA Nephropathy

No longer recruiting at 6 trial locations

Overseen By Ionis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ionis Pharmaceuticals, Inc.

Must not be taking: Immunosuppressives

Disqualifiers: Diabetes, Lupus, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new treatment called IONIS-FB-LRx for individuals with primary IgA nephropathy, a kidney condition. The treatment targets a specific part of the immune system to potentially reduce kidney inflammation and damage. Suitable candidates for this trial have been diagnosed with IgA nephropathy and experience blood in their urine (hematuria) and excess protein in their urine (proteinuria). As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in kidney disease treatment.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop your current medications, but it does exclude those who have taken immunosuppressive or immunomodulatory medications in the past 12 months, except for short-term treatments. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that IONIS-FB-LRx is likely to be safe for humans?

Research has shown that IONIS-FB-LRx is generally safe and well-tolerated in people with IgA nephropathy. Participants in studies did not report any major safety concerns. The treatment met its main goals and had positive effects, such as reducing protein in urine, which is a promising indicator. This suggests that IONIS-FB-LRx is generally well-tolerated by those who participated in previous studies. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for IgA nephropathy, which often includes treatments like corticosteroids and immunosuppressants, IONIS-FB-LRx works differently by targeting the complement system, specifically the factor B protein. This new approach aims to reduce inflammation and damage to the kidneys more directly and precisely. Researchers are excited because this mechanism could offer a more targeted treatment with potentially fewer side effects compared to broad immune suppression. This specificity could lead to improved kidney function and better outcomes for patients with IgA nephropathy.

What evidence suggests that IONIS-FB-LRx might be an effective treatment for IgA nephropathy?

Research shows that IONIS-FB-LRx, the treatment under study in this trial, holds promise for treating IgA nephropathy, a kidney disease. In earlier studies, this treatment reduced protein in the urine by an average of 44% after six months. Lowering protein levels in urine is crucial for improving kidney health in people with IgA nephropathy. The treatment targets a protein called complement factor B, part of the immune system that can harm the kidneys. Overall, the consistent positive results suggest that IONIS-FB-LRx could be effective for people with this condition. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

Adults with confirmed primary IgA nephropathy, a kidney disease marked by blood and protein in urine, can join this trial. They must not be pregnant or breastfeeding and should use effective birth control if necessary. People with recent major health events, certain immune deficiencies, severe infections, very low kidney function, or other specific kidney diseases cannot participate.

Inclusion Criteria

You have been diagnosed with primary immunoglobulin A (IgA) nephropathy through a biopsy.

Presence of blood in the urine

Presence of protein in the urine.

Exclusion Criteria

Treatment with another investigational drug, biological agent, or device within 6 months of screening, or 5 half-lives of investigational agent, whichever is longer

I haven't taken long-term immune system medications in the last year.

I have not had any infections in the last 30 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive IONIS-FB-LRx for 24 weeks to evaluate its effectiveness and safety

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Optional Treatment Extension

Participants may opt into an additional 48 weeks of treatment with IONIS-FB-LRx

48 weeks

What Are the Treatments Tested in This Trial?

Interventions

• IONIS-FB-LRx

Trial Overview The trial is testing IONIS-FB-LRx's ability to safely treat IgA nephropathy by inhibiting complement factor B. It will also assess how the drug affects plasma factor B levels and serum activity related to the body's defense (complement) system.

How Is the Trial Designed?

Group I: IONIS-FB-LRx Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD

Citations

1. ir.ionis.com ir.ionis.com/news-releases/news-release-details/ionis-presents-positive-phase-2-data-patients-iga-nephropathy

Ionis presents positive Phase 2 data in patients with IgA ... IONIS-FB-LRx achieved a 44% mean reduction in proteinuria in patients treated for 6 months. – Roche to advance IONIS-FB-LRx into Phase 3 development in H1 ...

2. journals.lww.com journals.lww.com/jasn/fulltext/2024/10001/phase_ii_results_of_an_investigational_rna.1525.aspx

Phase II Results of an Investigational RNA Therapeutic to... Conclusion: IONIS-FB-L Rx met primary endpoints in patients with biopsy-confirmed IgAN. This Ph2 open-label study provides consistent clinical evidence ...

3. asn-online.org asn-online.org/education/kidneyweek/2024/program-abstract.aspx?controlId=4167906

Kidney Week IONIS-FB-LRx met primary endpoints in patients with biopsy-confirmed IgAN. This Ph2 open-label study provides consistent clinical evidence supporting ongoing ...

4. ir.ionis.com ir.ionis.com/news-releases/news-release-details/ionis-partner-licenses-rare-kidney-disease-treatment-and-will

Ionis partner licenses rare kidney disease treatment and ... Positive data from a Phase 2 study of IONIS-FB-LRx support further development for treatment of patients with IgA nephropathy.

5. prnewswire.com prnewswire.com/news-releases/ionis-presents-positive-phase-2-data-in-patients-with-iga-nephropathy-at-american-society-of-nephrologys-kidney-week-2022-301669838.html

Ionis presents positive Phase 2 data in patients with IgA ... IONIS-FB-LRx achieved a 44% mean reduction in proteinuria in patients treated for 6 months. – Roche to advance IONIS-FB-LRx into Phase 3 ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S2468024924003929

A GLOBAL PHASE 3 TRIAL OF RO7434656, AN ... IMAGINATION aims to evaluate the efficacy and safety of RO7434656 in adults with IgAN using a broad range of assessments over 105 weeks.