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Study on Long-Term Safety and Effects of Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

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 disease called Atypical Hemolytic Uremic Syndrome (aHUS). This condition affects the blood and kidneys, leading to problems like low platelet counts, destruction of red blood cells, and kidney failure. The treatment being tested in this study is a medication called Iptacopan, which is taken as hard gelatin capsules. The purpose of the study is to evaluate the long-term safety and tolerability of Iptacopan in individuals with aHUS who have already participated in a previous study involving this medication.

Participants in this study will continue taking Iptacopan to see how well it works over a longer period. The study will monitor the safety of the medication by checking for any side effects and changes in health, such as blood tests and heart monitoring. The study will also look at how well the medication helps manage the symptoms of aHUS, like maintaining healthy blood counts and kidney function, without the need for other treatments.

The study is designed to last several years, allowing researchers to gather comprehensive data on the effects of Iptacopan over time. Participants will be regularly assessed to ensure their safety and to track the effectiveness of the treatment in managing aHUS symptoms. This research aims to provide valuable information on the long-term use of Iptacopan for people living with aHUS.

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, ensure that the informed consent form is signed. This document confirms your understanding and agreement to participate in the study.

    Verify that you have completed a previous phase 3 study involving iptacopan and are currently benefiting from the treatment, as assessed by the study doctor.

  2. Step 2

    Vaccination check

    Ensure that your vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae are up to date. This includes receiving any necessary booster shots according to local health guidelines.

  3. Step 3

    Medication administration

    You will continue taking iptacopan in the form of hard gelatin capsules. The medication is taken orally.

    Follow the dosage and frequency as prescribed by the study doctor. The duration of administration will be determined by the study protocol and your response to the treatment.

  4. Step 4

    Safety evaluations

    Throughout the study, regular safety evaluations will be conducted. These include monitoring for any adverse events or serious adverse events, checking safety laboratory parameters, vital signs, and performing electrocardiograms (ECGs).

  5. Step 5

    Monitoring health outcomes

    Your health will be monitored for the absence of thrombotic microangiopathy (TMA) manifestations without the use of anti-C5 antibody therapy. TMA is a condition that affects blood cells and kidneys.

    The study will assess your kidney function and other health parameters to ensure the treatment's effectiveness and safety.

  6. Step 6

    Study duration

    The study is expected to continue until April 15, 2029. Your participation will involve regular visits and assessments as outlined in the study schedule.

Who can join the trial?

6 criteria

  • Signed informed consent must be obtained before participating in the study. This means you agree to join the study after understanding all the details.
  • You must be willing and able to follow the study's schedule and activities.
  • You should have completed the full treatment period of a previous Novartis-sponsored study for aHUS and still be receiving the study treatment called iptacopan, which is considered beneficial by the study doctor.
  • Your vaccinations against certain infections should be up to date. These infections include Neisseria meningitidis (a type of bacteria that can cause serious infections), Streptococcus pneumoniae (another type of bacteria that can cause pneumonia and other infections), and Haemophilus influenzae (a bacteria that can cause various infections).
  • Both males and females can participate in the study.
  • The study includes participants from vulnerable populations, which means it considers people who might need special attention or care.

Who cannot join the trial?

10 criteria

  • Patients with any other serious medical conditions that might interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial within the last 30 days.
  • Patients with a history of allergic reactions to the study medication or similar drugs.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients with a history of drug or alcohol abuse.
  • Patients with certain infections that could affect the study results.
  • Patients who have received certain medications that might interfere with the study.
  • Patients with a history of cancer, except for some types of skin cancer.
  • Patients with uncontrolled high blood pressure.
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Investigated drugs

Iptacopan is a medication being studied for its long-term safety and effectiveness in people with a condition called atypical hemolytic uremic syndrome (aHUS). This condition affects the blood and kidneys, leading to problems like low red blood cell counts and kidney damage. Iptacopan works by targeting a specific part of the immune system that is thought to play a role in causing these problems. The goal of using iptacopan in this study is to see if it can help manage the symptoms of aHUS over a long period, making it easier for patients to live with this condition.

What is already known about the treatment

Iptacopan – Iptacopan is administered orally in the form of a pill. It is currently being studied in clinical trials to assess its long-term safety and effectiveness for treating atypical Hemolytic Uremic Syndrome (aHUS), a rare disease that affects the blood and kidneys. The medication works by inhibiting a specific protein in the immune system, which helps prevent the destruction of red blood cells and improves kidney function. Iptacopan is classified as a complement inhibitor, a type of drug that targets the immune system to reduce inflammation and tissue damage.

Investigated diseases

Atypical Hemolytic Uremic Syndrome (aHUS) – Atypical Hemolytic Uremic Syndrome is a rare disease characterized by the formation of small blood clots in the blood vessels, leading to the destruction of red blood cells. This process can cause a decrease in platelet count, known as thrombocytopenia, and result in hemolytic anemia, where red blood cells are destroyed faster than they can be made. The disease often affects the kidneys, leading to impaired kidney function and potentially causing acute kidney injury. Over time, the continuous formation of blood clots can lead to chronic kidney damage. The condition is associated with abnormalities in the regulation of the complement system, a part of the immune system. It can manifest with symptoms such as fatigue, high blood pressure, and reduced urine output.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502965-34-00Protocol codeCLNP023F12001BEstimated enrolment80 patientsSponsorNovartis Pharma AG

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