Iptacopan for IgA Nephropathy

Not yet recruiting at 2 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Must be taking: ACE inhibitors, ARBs

Must not be taking: Immunosuppressants, Corticosteroids

Disqualifiers: Secondary IgAN, IgA vasculitis, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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: Unknown

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests iptacopan, a new treatment for children with IgA nephropathy (IgAN), a kidney disease that can cause kidney damage. The researchers aim to determine if iptacopan can reduce protein in urine, a sign of kidney issues, and to assess how the body processes the drug and its safety. Children who have had a kidney biopsy showing IgAN and still exhibit high protein levels in their urine despite other treatments might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for IgAN.

Will I have to stop taking my current medications?

Participants must have been on a stable dose of ACE inhibitors or ARBs for at least 120 days before starting the study drug. If you are taking other medications for IgA nephropathy, like diuretics or SGLT2 inhibitors, those doses should also be stable for at least 120 days before the trial begins.

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

Research has shown that iptacopan is generally safe for patients with IgA nephropathy. In studies, only a small number of patients (2.7%) discontinued its use due to side effects. The infection rate among those taking iptacopan was similar to those not taking it, indicating it does not significantly increase infection risk. These findings suggest that iptacopan is reasonably safe for individuals with this condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for IgA nephropathy?

Unlike the standard treatments for IgA nephropathy, which typically include steroids and immunosuppressants, iptacopan offers a novel approach by specifically targeting the alternative complement pathway. Researchers are excited about iptacopan because it acts on a protein called factor B, potentially reducing kidney inflammation and damage more directly. This targeted mechanism could result in fewer side effects compared to existing options, providing a safer and more effective treatment for patients.

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

Research shows that iptacopan, the investigational treatment in this trial, helps reduce proteinuria, which is the presence of excess protein in the urine, in patients with IgA nephropathy, a type of kidney disease. Studies have demonstrated that this treatment can significantly lower protein levels in the urine, which is crucial because high levels can lead to kidney failure. Additionally, iptacopan reduces hematuria, or blood in the urine, more effectively than a placebo. These improvements suggest that iptacopan can slow kidney damage in people with IgA nephropathy. The treatment also has a similar rate of side effects as a placebo, indicating it is generally safe. ^{1 3 5 6 7}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for pediatric patients aged 2 to <18 with primary IgA Nephropathy (IgAN), proven by kidney biopsy. Participants must weigh at least 35 kg, have received specific vaccinations, and show proteinuria despite treatment with ACE inhibitors or ARBs. They should not have severe kidney damage and must have stable organ function.

Inclusion Criteria

I weigh at least 35 kg.

I am between 2 and 17 years old.

My kidney biopsy confirms IgAN with less than 50% fibrosis and less than 25% crescents.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 11 weeks

Run-in

Baseline assessments including two UPCR FMV measurements are obtained

2 weeks

Treatment

Participants receive iptacopan to assess proteinuria reduction, pharmacokinetics, safety, and tolerability

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 week

Rollover Extension Program (optional)

Participants may enroll in a program to continue receiving open-label iptacopan

What Are the Treatments Tested in This Trial?

Interventions

• Iptacopan

Trial Overview The study tests iptacopan's ability to reduce protein in urine, its safety, how the body processes it, and tolerability in children with IgAN. It's an open-label Phase III trial where all participants receive iptacopan without being compared to a placebo or other drug.

How Is the Trial Designed?

Group I: iptacopan Experimental Treatment 1 Intervention

Participants in Cohort 1 (12 to \< 18 years old) will receive iptacopan at the dose of 200 mg twice per day. Participants in Cohort 2 (2 to \< 12 years old) will receive iptacopan at a dose tbd.

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. novartis.com novartis.com/news/media-releases/new-novartis-fabhalta-iptacopan-data-show-clinically-meaningful-and-statistically-significant-proteinuria-reduction-383-versus-placebo-patients-iga-nephropathy-igan

New Novartis Fabhalta® (iptacopan) data show clinically ... New Novartis Fabhalta® (iptacopan) data show clinically meaningful and statistically significant proteinuria reduction of 38.3% versus placebo ...

7. kireports.org kireports.org/article/S2468-0249(24)02385-4/fulltext

WCN25-799 EFFICACY AND SAFETY OF IPTACOPAN IN ... Iptacopan was well tolerated (treatment was discontinued due to AEs in 2.7% patients in each arm) and the infection rate in the iptacopan arm ...