Active not recruitingPHASE3INTERVENTIONAL

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

This study is designed for individuals living with a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). It's an important study focusing on a medicine called Danicopan, which is used alongside another PNH treatment called a C5 inhibitor (specifically eculizumab or ravulizumab). The main goal is to see how safe and effective Danicopan is over a longer period when added to your current treatment. If you've previously taken part in an Alexion study involving Danicopan as an add-on to your C5 inhibitor, this study offers a chance to continue receiving the medication while helping researchers understand its long-term effects. The study will last for up to three years, and you'll continue to receive your regular PNH treatment alongside Danicopan.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

Start

28 Oct 2022

Estimated completion

31 Jul 2026

Paroxysmal Nocturnal Hemoglobinuria

What is this study about?

This research study is looking at a medicine called Danicopan. It's designed for people who have a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, often shortened to PNH. PNH is a condition where your red blood cells are attacked by a part of your immune system, which can lead to problems like anaemia (low red blood cells) and blood clots. Some people with PNH are already being treated with medicines called C5 inhibitors, such as eculizumab or ravulizumab, which help to control the condition.

In this study, Danicopan is given as an additional treatment on top of your current C5 inhibitor medicine. The main aim is to discover how well Danicopan works and how safe it is over a longer period when used alongside your existing PNH treatment. This is important because it helps doctors understand the potential long-term benefits and any side effects of adding Danicopan to the current ways we treat PNH.

Because this is a 'long-term extension' study, it’s specifically for people who have already completed a previous study where they received Danicopan with their C5 inhibitor. This allows researchers to gather more information on the medicine's effects over several years, which is crucial for understanding its full impact on PNH patients.

Key takeaways

This study is for people with PNH already taking a C5 inhibitor.
It's exploring the long-term safety and effectiveness of Danicopan as an add-on treatment.
Participation lasts up to three years with regular check-ups.
You must have been in a previous Danicopan study to be eligible.
Your current C5 inhibitor treatment will continue as normal.
There's a chance to continue receiving the study drug for longer.

Who may be eligible?

To join this study, you must have already taken part in a previous Alexion study where you received Danicopan alongside your C5 inhibitor treatment for PNH. You also need to be able to understand the study information and sign a consent form, agreeing to follow the study's rules.

You'll need to make sure your vaccinations for a specific type of meningitis (Neisseria meningitidis) are up-to-date, following UK guidelines for people taking medicines that affect the immune system.

There are some reasons why you might not be able to join. For example, if you have other serious health problems that your doctor thinks could make it unsafe for you to participate, or make it hard to accurately assess the study's results. Also, if you had to stop taking Danicopan in the previous study for health reasons (not just to join this long-term study), you won't be able to participate. Pregnant or breastfeeding women, or those planning to get pregnant during the study, also cannot take part.

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.

Have I previously completed an Alexion study with Danicopan for PNH?
Am I currently taking eculizumab or ravulizumab for PNH?
Am I able to understand the study details and sign a consent form?
Are my meningitis vaccinations up-to-date, or am I willing to get them?
Am I not pregnant, breastfeeding, or planning to conceive during the study?
Does my doctor think I am healthy enough to participate safely?

Answer every question to see your result.

What does participation involve?

If you join this study, you will continue to receive Danicopan as an add-on to your existing PNH medication (either eculizumab or ravulizumab). The total study length will be up to three years. Throughout this time, you will have regular visits with the study team. During these visits, there will be various assessments, which might include blood tests, physical examinations, and discussions about how you are feeling and any side effects you might be having. These assessments are important for the study doctors to monitor your health, how the medicines are working, and to ensure your safety. You will need to complete all the required checks and appointments from your previous study before starting this new one.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit of continuing to receive Danicopan, which could help manage your PNH symptoms and improve your quality of life. However, like all medicines, Danicopan can have side effects, and some risks are not yet fully known, especially with long-term use. The study team will monitor you closely for any side effects or changes in your health. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (45)

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

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Los Angeles, United States
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Chicago, United States
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Baltimore, United States
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Kansas City, United States
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Long Island City, United States
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Cleveland, United States
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Dallas, United States
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Porto Alegre, Brazil
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Rio de Janeiro, Brazil
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Toronto, Canada
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Brno, Czechia
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Lille, France

Common questions

What is PNH?

PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare condition where red blood cells are destroyed too quickly, which can cause symptoms like tiredness and discomfort.

What is Danicopan?

Danicopan is a medicine being studied as an additional treatment for PNH, used alongside other PNH medicines called C5 inhibitors.

Will I stop my current PNH medication?

No, you will continue to take your current PNH medication (either eculizumab or ravulizumab) throughout this study, with Danicopan added on top.

How long will the study last?

The study will last for up to three years, allowing researchers to fully understand the long-term effects of Danicopan.

What if I change my mind about taking part?

You have the right to leave the study at any time without having to give a reason, and it won't affect your medical care.