Iptacopan for IgA Nephropathy

Recruiting at 3 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Must be taking: ACEi, ARB

Must not be taking: Immunosuppressants, Corticosteroids

Disqualifiers: Secondary IgAN, HIV, Hepatitis, 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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 3

Duration: 9 months

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called iptacopan for individuals with IgA nephropathy, a kidney disease where the immune system harms the kidneys. The researchers aim to assess iptacopan's effects by measuring changes in kidney function and damage over nine months. Participants must have confirmed IgA nephropathy and should be on stable medication for blood pressure and kidney protection, such as ACE inhibitors or ARBs, for about three months before starting the trial. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in treatment.

Will I have to stop taking my current medications?

Participants must continue taking ACE inhibitors or ARBs at a stable dose throughout the study. If you are on other medications like diuretics or SGLT2 inhibitors, those doses should also be stable for at least 90 days before starting the trial. However, certain medications like immunosuppressive agents and herbal treatments for IgAN are not allowed.

Is there any evidence suggesting that iptacopan is likely to be safe for humans?

A previous study found no unexpected safety issues with iptacopan. The number of unwanted effects matched expectations. Research also showed that side effects related to the treatment occurred in about the same number of patients as those taking a placebo (a pill with no active medicine). This suggests that iptacopan is generally well-tolerated. However, these findings are based on earlier studies and may vary for each individual. Always consult a healthcare professional to understand what joining a trial might mean. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for IgA nephropathy focus on controlling blood pressure and reducing proteinuria, often using ACE inhibitors or ARBs. But iptacopan works differently, targeting a part of the immune system known as the complement pathway. Researchers are excited about iptacopan because it specifically inhibits factor B, a key protein in this pathway, potentially reducing kidney inflammation and damage more effectively. This targeted approach could offer better protection for the kidneys compared to standard therapies.

What evidence suggests that iptacopan might be an effective treatment for IgA Nephropathy?

Research has shown that iptacopan can greatly reduce the amount of protein in the urine of patients with IgA nephropathy (IgAN). Lowering protein levels in urine is crucial because high levels can harm the kidneys. Studies have also found that iptacopan can slow the progression of kidney function decline. Additionally, it helps reduce blood in the urine, another symptom of IgAN. These findings suggest that iptacopan may effectively manage IgAN by protecting the kidneys and reducing symptoms. All participants in this trial will receive iptacopan, allowing further evaluation of its effectiveness in treating IgAN. ^{2 4 5 6 7}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

Adults with IgA Nephropathy (IgAN) who have protein in their urine and confirmed diagnosis by kidney biopsy. They must be vaccinated against certain infections or take antibiotics, able to give informed consent, communicate well, and follow study requirements. Participants should be on stable doses of specific blood pressure medicines for about 90 days before the trial.

Inclusion Criteria

I have been vaccinated against meningitis and pneumonia at least 2 weeks before starting the study treatment, or I will start antibiotics if I need to begin treatment sooner.

I have signed the consent form and can follow the study's requirements.

My protein levels in urine are high.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Baseline

Baseline visit with kidney biopsy performed

1 day

1 visit (in-person)

Treatment

Participants receive iptacopan 200 mg b.i.d for a 9-month treatment period

9 months

Monthly visits (in-person)

Follow-up

Follow-up biopsy performed at the end of study to assess changes

1 day

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Iptacopan

Trial Overview The trial is testing Iptacopan's effect on IgAN when added to standard care. It looks at changes in kidney tissue and disease markers over 9 months to understand how it affects immune responses and kidney health.

How Is the Trial Designed?

Group I: Iptacopan Experimental Treatment 1 Intervention

All the study participants will receive iptacopan 200 mg oral capsule b.i.d, while remaining on the maximally tolerated or locally approved maximal daily doses of ACEi/ARB throughout the treatment period.

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1. nejm.org nejm.org/doi/10.1056/NEJMoa2410316

Alternative Complement Pathway Inhibition with Iptacopan ... Among patients with IgA nephropathy, treatment with iptacopan resulted in a significant and clinically meaningful reduction in proteinuria as ...

2. novartis.com novartis.com/news/media-releases/novartis-fabhalta-iptacopan-meets-phase-iii-primary-endpoint-slows-kidney-function-decline-patients-iga-nephropathy-igan

Novartis Fabhalta® (iptacopan) meets Phase III primary ... Up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring dialysis or ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S2468024925006618

Iptacopan in patients with IgA nephropathy from East Asia Iptacopan led to a greater improvement in hematuria than placebo. Treatment-emergent adverse events occurred in a similar proportion of patients ...

4. medcommshydhosting.com medcommshydhosting.com/CRM/Renal//ASN/presentations/2024/posters/ASN2024_Safety_and_efficacy_of_iptacopan_Poster.pdf

Safety and Efficacy of Iptacopan in Patients with IgA ... INTRODUCTION. • IgA nephropathy (IgAN) is the most common primary glomerulonephritis worldwide with a global incidence of. 2.5/100,000/year.1. • Iptacopan ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04578834

NCT04578834 | Study of Efficacy and Safety of LNP023 in ... The purpose of the study is to evaluate the efficacy and safety of iptacopan (LNP023) compared to placebo on proteinuria reduction and slowing disease ...

6. nejm.org nejm.org/doi/abs/10.1056/NEJMoa2410316

Alternative Complement Pathway Inhibition with Iptacopan ... There were no unexpected safety findings with iptacopan. The incidence of adverse events that occurred during the treatment period was similar ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10166738/

Design and Rationale of the APPLAUSE-IgAN Study APPLAUSE-IgAN will evaluate the benefits and safety of iptacopan, a novel targeted therapy for IgAN, in reducing complement-mediated kidney damage.