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Study on the Safety and Effects of Iptacopan in Children with Paroxysmal Nocturnal Hemoglobinuria (PNH) Aged 2 to 17 Years

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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) in children and teenagers aged 2 to under 18 years. The study will use a medication known as iptacopan hydrochloride, which is taken as hard gelatin capsules. The purpose of the study is to understand how the body processes this medication and to check its safety and how well it is tolerated by the participants.

Participants in the study will take iptacopan hydrochloride for a period of 26 weeks. During this time, researchers will monitor the participants to ensure the medication is safe and to observe any side effects. The study will also look at how the medication affects the levels of hemoglobin, a protein in red blood cells that carries oxygen, and other important health markers. Some participants may be switching from another treatment, while others may be new to this type of medication.

The study will not involve any placebo treatments. Participants will receive the actual medication throughout the study. The goal is to gather information that could help improve treatment options for children and teenagers with Paroxysmal Nocturnal Hemoglobinuria in the future. The study is expected to continue until 2031, allowing researchers to collect comprehensive data over time.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participants will be informed about the trial's purpose, which is to assess the safety and tolerability of the medication called iptacopan hydrochloride in children and teenagers with a condition known as paroxysmal nocturnal hemoglobinuria (PNH).

  2. Step 2

    Vaccination requirements

    Participants must be vaccinated against certain infections, including Neisseria meningitidis and Streptococcus pneumoniae, at least two weeks before starting the study medication. If not previously vaccinated, or if a booster is needed, the vaccine should be administered according to local guidelines. If the study treatment needs to start earlier than two weeks after vaccination, preventive antibiotic treatment should be initiated.

    Vaccination against Haemophilus influenzae is also recommended at least two weeks before starting the medication.

  3. Step 3

    Medication administration

    Participants will take iptacopan hydrochloride in the form of hard gelatin capsules by mouth. The specific dosage and frequency will be determined by the study team based on individual needs and conditions.

  4. Step 4

    Treatment period

    The treatment period will last for 26 weeks. During this time, participants will be monitored for any side effects and changes in their health. Regular check-ups will be conducted to ensure the safety and effectiveness of the treatment.

  5. Step 5

    Safety evaluations

    Throughout the study, safety evaluations will be conducted. These include monitoring for any adverse effects, checking laboratory parameters, vital signs, and cardiovascular health.

  6. Step 6

    End of treatment

    At the end of the 26-week treatment period, participants will undergo a final evaluation to assess the overall impact of the medication on their condition and health.

Who can join the trial?

9 criteria

  • Participants must be male or female and between the ages of 2 and 17 years old.
  • Participants must have a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), confirmed by a special blood test called high-sensitivity flow cytometry. This test checks for certain types of blood cells.
  • Participants must have a clone size of certain white blood cells (granulocytes/monocytes) that is 10% or more.
  • Participants in Cohort 1 must weigh at least 35 kg (about 77 pounds).
  • If participants are already being treated with anti-C5 therapy (a type of medication), they must have been on a stable dose for at least 6 months before joining the study.
  • Participants who have not been treated with anti-C5 therapy before must have a hemoglobin level (a measure of red blood cells) of less than 10 g/dL, confirmed by a central laboratory test during screening.
  • Participants who have not been treated with anti-C5 therapy before must have a lactate dehydrogenase (LDH) level that is more than 1.5 times the upper limit of normal, confirmed by at least two laboratory tests 2 to 6 weeks apart during the screening period.
  • Participants must be vaccinated against Neisseria meningitidis and Streptococcus pneumoniae infections before starting the study treatment. If not previously vaccinated, or if a booster is needed, the vaccine should be given at least 2 weeks before the first study drug is administered. If the study treatment needs to start earlier, preventive antibiotic treatment should be started.
  • It is recommended that participants be vaccinated against Haemophilus influenzae at least 2 weeks before starting the study treatment, according to local guidelines.

Who cannot join the trial?

8 criteria

  • Patients with a history of **allergic reactions** to the study medication cannot participate. An allergic reaction is when your body reacts badly to something, like a rash or trouble breathing.
  • Patients who are currently taking other medications that might interfere with the study drug are not eligible. This means if you are on certain medicines that could affect how the study drug works, you cannot join.
  • Patients with **severe liver or kidney disease** cannot participate. This means if your liver or kidneys, which help clean your blood, are not working well, you cannot join.
  • Patients who are pregnant or breastfeeding are not eligible. This means if you are expecting a baby or feeding a baby with breast milk, you cannot join.
  • Patients with a history of certain types of **cancer** cannot participate. Cancer is when cells in your body grow in a way that is not normal.
  • Patients who have participated in another clinical trial within the last 30 days are not eligible. This means if you were part of another study recently, you cannot join this one.
  • Patients with **uncontrolled high blood pressure** cannot participate. High blood pressure is when the force of your blood against your artery walls is too high, and uncontrolled means it is not being managed well.
  • Patients with **active infections** that require treatment cannot participate. An active infection is when germs are causing illness in your body, and you need medicine to get better.
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Investigated drugs

Iptacopan is a medication being studied for its effects on a condition called paroxysmal nocturnal hemoglobinuria (PNH) in children and teenagers. PNH is a rare blood disorder that can cause red blood cells to break down too early. This medication is being tested to see how safe it is for young patients and how well their bodies can handle it over a period of 26 weeks. The study also aims to understand how the medication moves through the body and how it is processed. By participating in this trial, researchers hope to learn more about how this medication can help manage PNH in younger patients.

What is already known about the treatment

Iptacopan – Iptacopan is administered orally, meaning it is taken by mouth in the form of a pill. It is currently being studied in clinical trials, specifically in a phase 3 study, to evaluate its safety and how it behaves in the body in children with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The main purpose of iptacopan is to help manage PNH by targeting a specific part of the immune system that is overactive in this condition. It works by blocking a protein that contributes to the destruction of red blood cells, which is a key problem in PNH. Iptacopan is classified as a complement inhibitor, which means it helps to control the immune system's complement pathway to prevent damage to blood cells.

Investigated diseases

Paroxysmal Nocturnal Hemoglobinuria (PNH) – Paroxysmal Nocturnal Hemoglobinuria is a rare blood disorder characterized by the destruction of red blood cells. This destruction occurs due to a defect in the surface proteins of the red blood cells, making them more susceptible to being broken down by the immune system. The breakdown of these cells leads to the release of hemoglobin into the bloodstream, which can cause dark-colored urine, especially noticeable in the morning. Over time, this condition can lead to anemia, fatigue, and other complications due to the loss of red blood cells. The disease can also cause blood clots, which may lead to further health issues. PNH progresses at varying rates in different individuals, with symptoms potentially worsening over time.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2024-515926-10-00Protocol codeCLNP023I12201Estimated enrolment12 patientsSponsorNovartis Pharma AG

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