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

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What is this study about?

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.

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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Careggi Florence Italy
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Centre Hospitalier Universitaire De Lille Lille France

Other Sites

Site Name City Country Status
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Geniko Nosokomeio Thessalonikis George Papanikolaou Thessaloniki Greece
AORN San Giuseppe Moscati Avellino Avellino Italy
Grande Ospedale Metropolitano Bianchi Melacrino Morelli Reggio Calabria Italy
Regione Del Veneto Azienda ULSS N 7 Pedemontana Bassano Del Grappa Italy
Hippokration Hospital Athens Greece
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Clinic De Barcelona Barcelona Spain
Fakultni Nemocnice Brno Brno Czechia
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Ugwlsqdifrqroo Cwubdrh Kkrrqhrpi Gdansk Poland
Uivhixrjxf Dtead Sfpvo Dr Rjwt Lu Snbmfeqy Rome Italy
Hamidxse Uunkyyxgkyfse Hthkxnya Tdmhm y Picass Ihodwacv Czjrfg dideijudhimeqknkw (oygv Badalona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Czechia Czechia
Not recruiting
02.05.2022
France France
Not recruiting
02.05.2022
Greece Greece
Not recruiting
02.05.2022
Italy Italy
Not recruiting
02.05.2022
Poland Poland
Not recruiting
02.05.2022
Spain Spain
Not recruiting
02.05.2022

Trial locations

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.

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 ID:
2023-504867-18-00
Protocol code:
ALXN2040-PNH-303
Trial Phase:
Therapeutic confirmatory (Phase III)

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