A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated with Danicopan in an Alexion-sponsored Clinical Study.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder where the immune system attacks and destroys red blood cells. Many people take standard medicines called C5 inhibitors, such as Soliris or Ultomiris, but some still suffer from anaemia or tiredness. Danicopan is an add-on treatment designed to help protect red blood cells more fully. This long-term extension study is open to adults who were already treated with danicopan in a previous Alexion trial. The goal is to track how well the medicine works over time and check for any side effects. Doctors will measure blood counts, watch for signs of red blood cell breakdown, and see whether participants still need blood transfusions. They will also look closely at how the treatment affects patients' daily energy levels and general quality of life.
At a glance
What is this study about?
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition that affects red blood cells. In healthy blood, cells have a special shield that keeps them safe from the body's own immune defences. In PNH, red blood cells lack this shield, causing the immune system to break them down too quickly. This can lead to severe anaemia, extreme fatigue, and the need for regular blood transfusions.
Modern treatments known as C5 inhibitors (such as Soliris or Ultomiris) have changed how PNH is managed by stopping much of this cell destruction. However, for some people, a different part of the immune system continues to break down cells in the spleen and liver. Danicopan is a newer add-on medicine that targets this specific problem, working alongside standard infusions to offer extra protection.
This trial is an extension study. It allows patients who already took danicopan in an earlier study to carry on receiving it for a longer duration. By following participants over an extended period, researchers hope to confirm whether danicopan keeps working well, reduces the need for transfusions, and remains safe for long-term use.
Key takeaways
- This trial is for adults with PNH who already took part in a previous danicopan study.
- Danicopan is taken alongside standard PNH infusions like Soliris or Ultomiris.
- The main aim is to check the long-term safety and effectiveness of the add-on treatment.
- Researchers will monitor blood counts, energy levels, and transfusion needs.
- Taking part is voluntary, and you can stop at any time.
Who may be eligible?
This study is only open to adults aged 18 and older who have already been diagnosed with PNH and took part in an earlier Alexion clinical trial testing danicopan.
Because this is an extension trial, people who have never taken danicopan in one of these specific previous trials cannot join. Both men and women can take part, provided they tolerated the medicine reasonably well in the earlier study.
Your study doctor will check your overall health and medical history to make sure it is still safe and appropriate for you to keep receiving the treatment alongside your usual C5 inhibitor therapy.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH).
- I was previously enrolled and treated in an Alexion clinical study of danicopan.
- I am currently receiving standard treatment with a C5 inhibitor (such as Soliris or Ultomiris).
- I am willing to attend regular hospital appointments for tests and check-ups.
What does participation involve?
If you take part, you will continue to receive your standard PNH treatment (such as regular infusions of Soliris or Ultomiris) while taking danicopan as an add-on medication.
You will attend regular clinic visits where the medical team will monitor your health closely. These visits will involve standard safety checks, including physical examinations, blood tests, and urine tests. The blood tests will check your red blood cell levels, markers of cell breakdown, and general organ function.
You will also be asked to fill out short quality-of-life questionnaires to track your energy and fatigue levels. The trial continues over a long-term follow-up period, and your research team will clearly explain the exact visit schedule and expected duration before you agree to continue.
Potential risks and benefits
Locations (6)
- —UnverifiedFrance
- —UnverifiedCzechia
- —UnverifiedGreece
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedPoland
Common questions
What is an extension study?
An extension study allows patients who completed a previous trial of an experimental medicine to continue receiving it while doctors collect long-term safety data.
Will I have to stop my current PNH infusion?
No. Danicopan is designed to be an add-on therapy, meaning you will continue receiving your regular C5 inhibitor infusions (such as Soliris or Ultomiris).
Do I have to pay for the study medicine?
No. All study-related medicines, tests, and monitoring are provided free of charge during the trial.
How often will I need blood tests?
Blood tests will be taken regularly during scheduled clinic visits to check your blood counts and monitor your safety.
Can I leave the study if I change my mind?
Yes. You can withdraw your consent at any point without it affecting your standard medical treatment or your relationship with your healthcare team.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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