What side effects are associated with this type of intervention?

Common treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH) include eculizumab and ravulizumab, which are complement inhibitors. Known side effects of these treatments include an increased risk of infections, particularly meningococcal infections, as well as headaches, back pain, and infusion-related reactions. For treatments similar to Iptacopan, a Factor B inhibitor, specific side effect data may not be as well-documented, but they are expected to share some of the risks associated with complement inhibition, such as susceptibility to infections and potential infusion-related reactions.

What prior FDA approvals exist?

The FDA has approved several treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH), primarily focusing on complement inhibition. Eculizumab (Soliris) and ravulizumab (Ultomiris) are monoclonal antibodies that inhibit the complement protein C5, preventing the destruction of red blood cells. These treatments are similar to Iptacopan, which is a Factor B inhibitor targeting a different part of the complement pathway. While Iptacopan is still under investigation, it represents a novel approach by inhibiting Factor B, potentially offering an alternative mechanism to control hemolysis in PNH patients.

What other types of research exist?

Promising novel alternatives to Iptacopan for PNH patients include eculizumab and ravulizumab, which are current standard treatments. Additionally, ongoing research and trials may reveal new options, but specific novel alternatives beyond these were not detailed in the provided context.

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Complement Inhibitor

Iptacopan for Paroxysmal Nocturnal Hemoglobinuria (APPULSE Trial)

Phase 3

Waitlist Available

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment.

Be older than 18 years old

Must not have

History of cancer of any part of the body within the past 5 years

History of stem cell transplantation or any solid organ transplantation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 168 days

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a new medication called iptacopan in adults with a blood disorder called PNH. It aims to see if iptacopan is safe and effective. Iptacopan works by stopping the immune system from attacking red blood cells. The medication has shown significant improvement in hemoglobin levels and reduction in hemolysis.

Who is the study for?

Adults over 18 with Paroxysmal Nocturnal Hemoglobinuria (PNH) who've been on a stable anti-C5 antibody treatment for at least 6 months can join. They must not have HIV, be pregnant, or have had recent infections or transfusions. Vaccinations against certain infections are required before starting the trial.

What is being tested?

The trial is testing iptacopan's effectiveness and safety in PNH patients switching from their current treatments (eculizumab/ravulizumab) to iptacopan. It's an open-label study where all participants receive iptacopan for 24 weeks.

What are the potential side effects?

While specific side effects of iptacopan aren't listed here, common ones may include symptoms like headache, nausea, or potential increased risk of infection due to immune system changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been vaccinated against meningitis and pneumonia before starting iptacopan.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had cancer in any part of my body in the last 5 years.

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I have had a stem cell or solid organ transplant.

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I have not had any serious infections, including COVID-19, in the last 14 days.

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I needed a blood transfusion in the last 6 months.

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I have had repeated serious infections like meningitis or pneumonia.

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I am not pregnant, breastfeeding, nor planning to become pregnant during the study.

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I have had a fever of 100.4°F or higher in the last week.

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I am HIV positive.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 168 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 168 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 168 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in Hb levels to demonstrate non-inferiority of iptacopan

Secondary study objectives

Change from baseline in Absolute Reticulocytes count (ARC) levels

Change in Hb levels to demonstrate superiority of iptacopan

Change in fatigue score using FACIT-F questionnaire

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Iptacopan treatment in adult PNH patientsExperimental Treatment1 Intervention

Subjects will receive iptacopan at a dose of 200 mg b.i.d. orally

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Iptacopan

2021

Completed Phase 1

~40

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Paroxysmal Nocturnal Hemoglobinuria (PNH) is commonly treated with complement inhibitors such as eculizumab and ravulizumab, which target the C5 component of the complement system to prevent the destruction of red blood cells. Iptacopan, a Factor B inhibitor, works by inhibiting the alternative pathway of the complement system, thereby reducing hemolysis and thrombosis. These treatments are crucial for PNH patients as they help manage symptoms, reduce the risk of life-threatening complications, and improve quality of life by preventing the excessive breakdown of red blood cells and associated complications.

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