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Study on the Long-term Safety and Effectiveness of Danicopan with Ravulizumab or Eculizumab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Verified siteRegistered drugNo 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 study is testing a treatment called danicopan, which is used as an additional therapy alongside another type of medication known as a Complement Component 5 Inhibitor (C5i). The C5i medications being used in this study include Ultomiris and Soliris. Danicopan is taken as a film-coated tablet, while Ultomiris and Soliris are given as solutions for infusion, which means they are administered directly into the bloodstream through a vein.

The purpose of this study is to understand the long-term safety of using danicopan as an add-on treatment for patients with PNH who have previously been treated with danicopan in another study. Participants will continue their treatment over a period of time, during which they will receive either danicopan with a C5i or a placebo. The study will monitor any side effects and changes in health, such as hemoglobin levels, which is a protein in red blood cells that carries oxygen, and other blood-related measurements.

Throughout the study, participants will have regular check-ups to track their health and any changes in their condition. The study aims to gather information on how well danicopan works when used with C5i medications and to ensure it is safe for long-term use. This research is important for improving treatment options for people living with PNH.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing a previous clinical study with danicopan as an add-on to a complement component 5 inhibitor (C5i).

    Ensure documentation of vaccination for Neisseria meningitidis according to national guidelines or local practice.

  2. Step 2

    Treatment phase

    Receive danicopan in the form of a film-coated tablet for oral use.

    Continue treatment with a C5i, either ravulizumab or eculizumab, both administered as a solution for infusion intravenously.

    The objective is to assess the long-term safety of danicopan as an add-on therapy.

  3. Step 3

    Monitoring and assessments

    Regular monitoring for treatment-emergent adverse events (TEAEs) and serious TEAEs.

    Assess changes in hemoglobin levels, reticulocyte count, and lactate dehydrogenase (LDH) over time.

    Evaluate the proportion of patients achieving specific hemoglobin and LDH targets.

    Monitor transfusion avoidance, fatigue scores, and quality of life scores using standardized questionnaires.

    Safety laboratory parameters are checked periodically.

  4. Step 4

    Completion of the study

    The study is estimated to end by May 2, 2025.

    Final assessments will be conducted to evaluate the overall safety and efficacy of the treatment.

Who can join the trial?

5 criteria

  • Participants must have a condition called Paroxysmal Nocturnal Hemoglobinuria, which is a rare blood disorder.
  • Participants must have completed a previous clinical study with a medication called danicopan used alongside another treatment known as a complement component 5 inhibitor (C5i).
  • Participants must have proof of vaccination against a bacteria called Neisseria meningitidis. This means they need to be vaccinated again according to national or local guidelines for vaccines used with treatments that affect the immune system.
  • Both males and females are eligible to participate.
  • The study includes individuals from vulnerable populations, which means people who might need extra protection or care.

Who cannot join the trial?

7 criteria

  • Patients who do not have Paroxysmal Nocturnal Hemoglobinuria cannot participate. This is a rare blood condition where red blood cells break down too early.
  • Patients who are not in the age range specified for the study cannot participate. The study is open to certain age groups only.
  • Patients who are not taking a complement component 5 inhibitor (C5i) cannot participate. This is a type of medication used to treat certain conditions.
  • Patients who are not eligible for the long-term safety study of the treatment cannot participate.
  • Patients who are not part of the specified clinical trial groups cannot participate.
  • Patients who are not male or female cannot participate, as the study includes both genders.
  • Patients who are not considered part of a vulnerable population cannot participate. Vulnerable populations may include groups like children, pregnant women, or those with certain health conditions.
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Investigated drugs

  • Danicopan

    is a medication being studied for its long-term safety and effectiveness when used as an additional treatment. It is specifically used in patients with a condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). Danicopan is added to another type of medication known as a complement component 5 inhibitor (C5i) to help manage the symptoms of PNH.

  • Complement Component 5 Inhibitor (C5i)

    is a type of medication that helps to prevent the breakdown of red blood cells in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). It works by blocking a part of the immune system that can cause damage to red blood cells. In this study, it is used in combination with Danicopan to see if the combination is safe and effective for long-term use.

What is already known about the treatment

Danicopan – Danicopan is administered orally and is currently being studied as an add-on therapy for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). It is in the clinical trial phase, focusing on its long-term safety and efficacy when used alongside a complement component 5 inhibitor. The main therapeutic indication for Danicopan is to help manage PNH, a rare blood disorder. At the molecular level, Danicopan works by inhibiting a specific enzyme in the complement pathway, which is part of the immune system, thereby reducing the destruction of red blood cells. It is classified pharmacologically as a complement factor D inhibitor.

Investigated diseases

Paroxysmal Nocturnal Hemoglobinuria – 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. As the condition progresses, patients may experience episodes of hemolysis, where red blood cells break down more rapidly, causing anemia and other complications. Over time, this can lead to an increased risk of blood clots and damage to vital organs. The disease is chronic and can vary in severity, with some individuals experiencing more frequent and severe symptoms than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-504867-18-00Protocol codeALXN2040-PNH-303Estimated enrolment80 patientsSponsorAlexion Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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