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Study on the Effectiveness and Safety of Iptacopan for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) Switching from Anti-C5 Antibody Treatment

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The trial will use a treatment called iptacopan, which is taken as hard gelatin capsules. Iptacopan is also known by its code name LNP023. The purpose of the study is to evaluate if iptacopan is as effective as the current standard treatment for PNH when patients switch to it.

Participants in the study will be adults who have been previously treated with an anti-C5 antibody, a type of medication used to manage PNH. The study will involve taking iptacopan orally twice a day. The trial will monitor changes in hemoglobin levels, which is a measure of the amount of red blood cells in the blood, over a period of time. This will help determine the effectiveness and safety of iptacopan for people with PNH.

The study is designed to last for a specific period, during which participants will be regularly assessed to track their response to the treatment. The goal is to see if iptacopan can maintain or improve hemoglobin levels in patients who switch from their current treatment. This trial is an important step in exploring new treatment options for individuals living with PNH.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initiation of iptacopan treatment

    Begin taking iptacopan, which is provided in the form of hard gelatin capsules.

    The medication is taken orally, twice daily.

    Ensure that vaccinations against Neisseria meningitidis, S. pneumoniae, and Haemophilus influenzae (if not previously received) are completed at least 2 weeks prior to starting treatment. If vaccinations are administered less than 2 weeks before starting iptacopan, a prophylactic antibiotic will be provided for at least 2 weeks after vaccination.

  2. Step 2

    Monitoring and assessment

    Regular monitoring of hemoglobin levels will occur throughout the trial.

    Hemoglobin levels will be specifically measured before starting iptacopan and then again at Day 126 and Day 168 to assess changes.

  3. Step 3

    Completion of trial

    The trial is expected to conclude by March 11, 2025.

    Final assessments will be conducted to evaluate the effectiveness and safety of iptacopan treatment.

Who can join the trial?

8 criteria

  • You must sign a form to show you understand and agree to take part in the study.
  • Both men and women who are **18 years or older** can participate.
  • You need to have a confirmed diagnosis of **Paroxysmal Nocturnal Hemoglobinuria (PNH)**, which is a specific blood condition.
  • You should have been on a stable treatment plan with certain medications (**anti-C5 antibody treatment**) for at least 6 months before joining the study. These medications include eculizumab or intravenous ravulizumab.
  • Your average **hemoglobin level** (a measure of red blood cells) should be **10 grams per deciliter (g/dL)** or higher, based on tests done over the past 6 months. You will also need two more tests during the screening period to confirm this level.
  • You must be vaccinated against certain infections, like **Neisseria meningitidis** and **S. pneumoniae**, before starting the new treatment. If you haven't been vaccinated yet, you need to get the vaccines at least 2 weeks before starting the treatment, unless the study doctor decides otherwise. If you start treatment less than 2 weeks after vaccination, you will need to take preventive antibiotics.
  • It is recommended to get vaccinated against **Haemophilus influenzae** infections if you haven't already, following local guidelines. This should also be done at least 2 weeks before starting the treatment, unless the study doctor decides otherwise. If you start treatment less than 2 weeks after vaccination, you will need to take preventive antibiotics.
  • You should be able to communicate well with the study doctor and understand and follow the study requirements.

Who cannot join the trial?

4 criteria

  • Patients who do not have **Paroxysmal Nocturnal Hemoglobinuria (PNH)**, a rare blood disorder, cannot participate.
  • Patients who are not within the specified age range for the trial cannot participate.
  • Patients who belong to certain clinical trial groups that are not included in this study cannot participate.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
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Investigated drugs

Iptacopan is an oral medication taken twice daily. It is being studied for its effectiveness and safety in adult patients with a condition called PNH (Paroxysmal Nocturnal Hemoglobinuria). The trial aims to determine if switching to iptacopan is as effective as the current standard treatment for patients who have already responded to anti-C5 antibody therapy.

What is already known about the treatment

Iptacopan – Iptacopan is administered orally in the form of tablets, taken twice daily. It is currently being studied in clinical trials for its effectiveness and safety in treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The main therapeutic indication for iptacopan is to manage PNH in patients who have shown a response to anti-C5 antibody treatments. At the molecular level, iptacopan works by inhibiting a specific protein in the complement system, which is part of the immune system, to prevent the destruction of red blood cells. It is classified pharmacologically as a complement inhibitor.

Investigated diseases

Paroxysmal Nocturnal Hemoglobinuria (PNH) – This is a rare blood disorder characterized by the destruction of red blood cells, leading to symptoms such as fatigue, shortness of breath, and dark-colored urine. The disease occurs due to a mutation in the bone marrow, causing red blood cells to be more susceptible to being broken down by the immune system. Over time, this can lead to anemia and other complications. PNH can also cause blood clots, which may affect various organs. The condition is chronic and can vary in severity among individuals. Regular monitoring of blood counts and symptoms is essential for managing the disease.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502148-10-00Protocol codeCLNP023C12303Estimated enrolment50 patientsSponsorNovartis Pharma AG

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