Iptacopan for Liver Disease

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Pregnancy, Arrhythmias, Investigational drugs, others

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

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

Duration: 11 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how the body processes a drug called iptacopan in individuals with varying levels of liver problems. The goal is to assess the safety and effectiveness of a single dose of the drug in these groups compared to those with healthy livers. Participants with mild, moderate, or severe liver issues who can stay in a research unit for the study are suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, it does exclude participants who have used other investigational drugs recently.

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

Research has shown that iptacopan, a treatment under study, has some known safety information. In studies for another condition, common side effects included headaches, sore throat, diarrhea, and stomach pain. Some participants also experienced bacterial or viral infections. Serious infections, such as those caused by certain bacteria, were less common but did occur.

These studies also reported some serious side effects, like kidney infections and urinary tract infections, though these were rare, affecting about 5% of participants. Notably, iptacopan is already approved for another condition, suggesting a level of safety, but it can still pose risks.

Prospective trial participants should consult a doctor about possible side effects. A doctor can help clarify what these risks might mean for individual cases. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Iptacopan is unique because it targets a specific part of the immune system known as the complement pathway, which plays a role in liver function and disease. Unlike other treatments for liver impairments that might focus on symptom management or general liver support, Iptacopan directly interferes with a part of the immune system that can cause inflammation and damage in the liver. Researchers are excited about Iptacopan because it could offer a more targeted approach, potentially leading to better outcomes for patients with varying degrees of liver impairment. This precision in targeting could mean fewer side effects and improved liver function in people who struggle with mild to severe hepatic issues.

What evidence suggests that iptacopan might be an effective treatment for hepatic impairment?

This trial studies iptacopan as a treatment that blocks factor B, a key component of the immune system's alternative complement pathway. Research has shown that iptacopan is a groundbreaking, powerful, and targeted oral medication. Early findings suggest that its concentration in the body remains stable, even in individuals with varying liver health. The trial includes groups for healthy participants and those with mild, moderate, or severe hepatic impairment, indicating that iptacopan might be effective regardless of liver condition. These early signs offer hope for its use in various conditions related to immune system issues. ^{6 7 8 9 10}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

Inclusion Criteria

Written approval was procured prior to any evaluation.

You possess the capacity to effectively converse with the investigator, comprehend and adhere to the study's demands.

You must be prepared to stay in the clinical research center as specified by the protocol.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Baseline Evaluation

Baseline evaluation conducted on Day -1 before the administration of the study drug

1 day

1 visit (in-person)

Treatment

Single dose administration of 200 mg of iptacopan followed by pharmacokinetic sampling

11 days

Daily visits for PK sampling

Follow-up

Participants are monitored for safety approximately 30 days after the last administration of the study drug

30 days

1 follow-up call

What Are the Treatments Tested in This Trial?

Interventions

• Iptacopan

How Is the Trial Designed?

Group I: Severe hepatic impairment patients Experimental Treatment 1 Intervention

Iptacopan 200 mg single dose

Group II: Moderate hepatic impairment patients Experimental Treatment 1 Intervention

Iptacopan 200 mg single dose

Group III: Mild hepatic impairment patients Experimental Treatment 1 Intervention

Iptacopan 200 mg single dose

Group IV: Healthy participants Experimental Treatment 1 Intervention

Iptacopan 200 mg single dose

Iptacopan is already approved in United States for the following indications:

Approved in United States as Fabhalta for:

• Paroxysmal nocturnal hemoglobinuria (PNH)
• Primary immunoglobulin A nephropathy (IgAN)

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. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11993288/

Effect of Target‐Mediated Disposition on Iptacopan Clinical ... This study assessed the effect of hepatic impairment (HI) on total (bound plus unbound) and unbound iptacopan pharmacokinetics (PK).

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05078580

NCT05078580 | Pharmacokinetic (PK) and Safety Study of ... This was an open-label, single dose parallel group study to evaluate the PK of iptacopan in participants with mild, moderate, and severe hepatic impairment ...

3. medcommshydhosting.com medcommshydhosting.com/CRM/Renal/WCN/presentations/2024/poster/Assessment_of_the_effect_of_hepatic_impairment_WCN24_poster.pdf

Assessment of the effect of hepatic impairment on iptacopan ... Iptacopan (LNP023) is an oral, first-in-class, highly potent, selective inhibitor of factor B, a key component of the alternative complement pathway1.

4. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2023/218276Orig1s000IntegratedR.pdf

218276Orig1s000 INTEGRATED REVIEW - accessdata.fda.gov Iptacopan binds directly to and inhibits factor B. A dedicated hepatic impairment study, CLNP023A2105, was conducted in subjects with normal hepatic function ...

5. novartis.com novartis.com/sg-en/sites/novartis_sg/files/Fabhalta-Feb2024.SIN-app060825-pdf.pdf

Core Data Sheet ... hepatic impairment, a negligible effect on the total systemic exposure of iptacopan was observed compared to subjects with normal hepatic function. Unbound ...

6. fabhalta.com fabhalta.com/pnh/safety-side-effects

Safety & Side Effects | PNH | FABHALTA® (iptacopan) Safety profile of FABHALTA · Adverse reactions reported in more than 5% of adults with PNH treated with FABHALTA during the initial study period (Weeks 0 to 24).

7. mayoclinic.org mayoclinic.org/drugs-supplements/iptacopan-oral-route/description/drg-20560921

Iptacopan (oral route) - Side effects & dosage Iptacopan may increase your chance of having serious infections, including Streptococcus pneumoniae or Neisseria meningitidis. Avoid people who ...

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2025/218276s004lbl.pdf

FABHALTA® (iptacopan) capsules, for oral use Serious adverse reactions included pyelonephritis, urinary tract infection and COVID-19. In APPOINT-PNH, serious adverse reactions were reported in 2 (5%) ...

9. fabhalta-hcp.com fabhalta-hcp.com/pnh/safety

Safety | PNH | FABHALTA (iptacopan) | HCP FABHALTA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria.

10. drugs.com drugs.com/sfx/iptacopan-side-effects.html

Iptacopan Side Effects: Common, Severe, Long Term The most common adverse reactions included headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, viral infection, nausea, rash, upper ...