RecruitingPHASE3INTERVENTIONAL

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

This study is for young people aged 12 to 17 who have a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). Specifically, it's for those already on standard treatments (ravulizumab or eculizumab) but still experiencing problems with their red blood cells breaking down too much, a condition called extravascular hemolysis. The main goal of the study is to find out if adding a new medicine, danicopan, to their current treatment can help improve their red blood cell levels (hemoglobin) over 12 weeks. Researchers want to see if this combination treatment can better manage their PNH symptoms. It's a Phase 3 study, meaning it's a later stage of testing, often comparing a new treatment to existing ones or a placebo to confirm its effectiveness and safety.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

Start

11 Aug 2025

Estimated completion

10 Mar 2028

Paroxysmal Nocturnal Hemoglobinuria PNH Extravascular Hemolysis

What is this study about?

This study is investigating a new medicine called danicopan for young people aged 12 to 17 who have a rare blood condition known as Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH causes red blood cells to break down too early, which can lead to anaemia (low red blood cell count) and other health issues. Many people with PNH are already treated with medicines like ravulizumab or eculizumab, which help stop red blood cells from breaking down inside the blood vessels.

However, some people still experience problems even with these treatments, specifically a condition called extravascular hemolysis. This means their red blood cells are still being destroyed, but in a different way – outside of the blood vessels. This can lead to ongoing anaemia and its related symptoms, like tiredness. This study wants to see if adding danicopan to the existing treatment can help improve these red blood cell levels and make people feel better.

The main aim is to check if danicopan increases the amount of haemoglobin in the blood, which is a measure of healthy red blood cells. By doing this, the researchers hope to find a better way to manage PNH for young people who aren't getting all the benefit they need from their current medication alone.

Key takeaways

This study is for young people (12-17) with PNH.
It tests a new add-on medicine, danicopan, to improve red blood cell levels.
Participants must already be using ravulizumab or eculizumab.
The main goal is to see if danicopan helps with ongoing red blood cell breakdown.
Regular health checks and blood tests will be part of the study.
You can stop participating at any time.

Who may be eligible?

To join this study, you must be between 12 and 17 years old and have a confirmed diagnosis of PNH. You should also be experiencing ongoing issues with red blood cells breaking down (extravascular hemolysis), even though you've been on either ravulizumab or eculizumab for at least 3 months, and your dose has been stable.

There are also some health requirements. For example, you need to have had certain vaccinations (for meningitis, Hib, and pneumonia) to protect you during the study. Your blood tests, especially for your red and white blood cells, liver function, and platelets, need to fall within certain limits. You also shouldn't have specific other health conditions like severe liver problems, certain bone marrow issues, or some types of infections.

It's important that you haven't had a major organ transplant or a bone marrow transplant in the past.

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.

Are you between 12 and 17 years old?
Have you been diagnosed with PNH?
Are you currently receiving ravulizumab or eculizumab for your PNH?
Have you had certain vaccinations (meningitis, Hib, pneumonia)?
Do you have ongoing anaemia or signs of red blood cells breaking down?
Do you have generally good health aside from your PNH?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve adding a new medication, danicopan, to your current treatment for PNH. You would continue to receive your usual ravulizumab or eculizumab. The study would closely monitor your health and how you respond to the new treatment over a period of time. This will likely involve regular hospital visits for blood tests and other checks to see how your red blood cell levels are changing.

You would have check-ups with the study team, who would ask about your symptoms and any side effects you might be experiencing. The total duration of the study would be outlined by the research team, but the main focus for evaluating the new medicine is 12 weeks. There may also be follow-up visits after this main period to continue monitoring your health.

Potential risks and benefits

There are potential benefits to taking part, such as receiving a new treatment that might improve your red blood cell levels and potentially reduce some of your PNH symptoms, like tiredness. You would also be closely monitored by a healthcare team. However, as with any new medication, there are potential risks and side effects that are not fully known yet, as danicopan is still being investigated. The study team will explain these in detail. It's crucial to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Research Site
Verified postcode
Saskatoon, Canada· Recruiting
Research Site
Verified postcode
Paris, France· Recruiting
Research Site
Verified postcode
Leeds, United Kingdom· Recruiting
Research Site
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is PNH?

PNH is a rare blood disorder where red blood cells break down too soon, which can cause anaemia and other problems.

What is 'extravascular hemolysis'?

This is when red blood cells are still breaking down, but in a different way, outside of your blood vessels, even if you're on PNH treatment.

What is danicopan?

Danicopan is an investigational medicine being tested as an add-on treatment for PNH to help improve red blood cell levels.

Will I stop my current PNH medication if I join?

No, you would continue your current ravulizumab or eculizumab and take danicopan in addition to it.

How long will the study last?

The main part of the study assessing the effectiveness of danicopan is 12 weeks, with potential for follow-up visits after that.

How to find out more

Alexion Pharmaceuticals, Inc. (Sponsor)

clinicaltrials@alexion.com 1-855-752-2356 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.