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Study on Long-Term Safety and Tolerability of Iptacopan for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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 the long-term safety and tolerability of a medication called iptacopan in patients with a rare blood disorder known as Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The medication being tested, iptacopan, is taken in the form of hard gelatin capsules and is designed to help manage this condition.

The purpose of the study is to evaluate how safe and well-tolerated iptacopan is when used over a long period. Participants in this study are individuals who have already completed previous phases of clinical trials with iptacopan. The study will monitor participants for any side effects or health changes while they continue taking the medication. This includes regular check-ups and assessments to ensure the medication is working as intended without causing harm.

Throughout the study, participants will be observed for any adverse events, which are unexpected health issues that may arise. The study will also track the stability of hemoglobin levels, which are important for carrying oxygen in the blood, and check for any need for blood transfusions. Additionally, the study will look for any occurrences of breakthrough hemolysis, which is when red blood cells break down too quickly, and any major vascular events, which are serious issues related to blood vessels. The study aims to provide valuable information on the long-term use of iptacopan for managing PNH.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment

    Participation begins after completing previous Phase II or Phase III studies with iptacopan.

    Eligibility requires a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) and being clinically stable on iptacopan for at least 3 months.

    Vaccinations against certain infections must be up to date.

  2. Step 2

    Treatment

    Receive iptacopan in the form of hard gelatin capsules.

    The medication is taken orally as prescribed by the study protocol.

    The objective is to evaluate the long-term safety and tolerability of iptacopan.

  3. Step 3

    Monitoring

    Regular safety evaluations are conducted, including monitoring for any adverse events.

    Laboratory tests and vital signs are checked periodically.

    The study aims to maintain hemoglobin levels and avoid blood transfusions.

  4. Step 4

    Follow-up

    Yearly follow-up intervals assess the absence of blood transfusions and occurrences of breakthrough hemolysis.

    Monitoring for Major Adverse Vascular Events (MAVE) is included in the follow-up.

  5. Step 5

    Completion

    The study is estimated to end by February 7, 2028.

    Final evaluations are conducted at the end of the study period.

Who can join the trial?

5 criteria

  • Participants must be **18 years or older**.
  • Participants must have a diagnosis of **Paroxysmal Nocturnal Hemoglobinuria (PNH)**, a rare blood condition.
  • Participants must have completed the treatment extension period of previous Phase II or any Phase III clinical studies with **iptacopan**, a medication being studied.
  • Participants must have received all necessary vaccinations against certain infections, including **Neisseria meningitidis**, **Streptococcus pneumoniae**, and **Haemophilus influenzae**. These are types of bacteria that can cause serious infections.
  • The study doctor must believe that the participant may benefit from continuing treatment with **iptacopan** and that the participant has been stable on this treatment for at least **3 months**.

Who cannot join the trial?

1 criterion

  • Participants who have not completed the treatment extension period of the previous Phase II and any Phase III clinical studies with iptacopan.
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Investigated drugs

Iptacopan is a medication being studied for its long-term safety and tolerability in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This trial is for patients who have already completed earlier phases of studies with iptacopan. The goal is to understand how well patients can tolerate the medication over an extended period and to monitor any potential side effects.

What is already known about the treatment

Iptacopan – Iptacopan is administered orally in the form of tablets. It is currently being studied in clinical trials, specifically in an open-label, multicenter roll-over extension program to assess its long-term safety and tolerability. The main therapeutic indication for iptacopan is Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. 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 PIGA gene, which affects the surface proteins that protect red blood cells from the immune system. Over time, the destruction of red blood cells can lead to anemia and other complications. Patients may experience episodes of hemolysis, where red blood cells break down more rapidly, often at night. The condition can also increase the risk of blood clots and other vascular issues. PNH is a chronic condition that requires ongoing monitoring and management.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-509843-28-00Protocol codeCLNP023C12001BEstimated enrolment193 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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