Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases

This study is for adults with primary immunodeficiency, a condition where the body's immune system doesn't work properly, making them prone to infections. Researchers are testing a new medicine called Newnorm, which is given as an injection under the skin. The main goal is to see how well Newnorm prevents serious bacterial infections like blood poisoning or severe pneumonia over a year. We'll also check how much of the medicine stays in the body and if it's safe to use. We'll look at other things too, like how often people get other types of infections, how quickly they recover, if they need antibiotics, and how their quality of life changes.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Octapharma Pharmazeutika Produktionsgesellschaft mbH

Enrolment target

Start

12 Jul 2024

Human Normal Immunoglobulin

What is this study about?

This study is about a new treatment called Newnorm, designed for people who have a condition called primary immunodeficiency. This means their immune system, which usually fights off germs, isn't working as well as it should. Because of this, people with primary immunodeficiency often get infections more easily and they can be more severe. Newnorm is a type of medicine called human immunoglobulin, which helps to boost the body's natural defences.

The main purpose of this study is to find out several important things about Newnorm. Firstly, we want to see how effective it is at preventing serious bacterial infections over a period of 52 weeks (about a year). This includes infections like blood poisoning (bacteraemia), meningitis (brain infection), bone infections (osteomyelitis), severe pneumonia, and internal abscesses. We will also be looking closely at how the body absorbs and uses Newnorm to make sure it's working as expected once it's in your system.

Beyond preventing serious infections, the study will also look at other benefits and effects. This includes how often people get other kinds of infections, like sinus problems or bronchitis, and how quickly those infections clear up. We'll also monitor how often antibiotics are needed, if hospital stays due to infection decrease, and if fever episodes are reduced. Importantly, the study will also assess how taking Newnorm affects a person's daily life, including time off work or school due to illness, and their overall quality of life.

Key takeaways

This study is testing a new under-the-skin injection called Newnorm.
It's for adults with primary immunodeficiency to help prevent infections.
The main goal is to see how well Newnorm reduces serious bacterial infections.
Researchers will also check for safety, other types of infections, and quality of life.
The study lasts for one year with regular check-ups.

Who may be eligible?

This study is looking for adults aged 18 and over who have primary immunodeficiency.

Both men and women can take part. The research team will need to make sure that Newnorm is suitable for you and that you meet all the health requirements for the study. They will check your medical history and current health to ensure it's safe for you to participate.

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 18 years old or older?
Do you have a diagnosis of primary immunodeficiency?
Are you generally willing to attend regular clinic visits?
Are you able to receive injections under your skin?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the Newnorm medicine as an injection under your skin. This is an 'open-label' study, which means both you and the study team will know you are receiving Newnorm. The study will last for 52 weeks (around one year). During this time, you will have regular clinic visits where the study team will check your health, ask about any infections you've had, and take blood samples to see how the medicine is working in your body. They will also ask you to fill out questionnaires about your quality of life. The team will provide you with all the necessary medication and support throughout the study, and closely monitor your health to ensure your safety.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of taking part in this study is receiving a new treatment, Newnorm, which may help to reduce the number and severity of infections you experience due to your primary immunodeficiency. This could lead to an improved quality of life. Potential risks might include side effects from the Newnorm injection, such as redness or soreness at the injection site, or other reactions. The study team will explain all known side effects and monitor you closely. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

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Unverified
Italy
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Unverified
Slovakia
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Unverified
Hungary
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Unverified
Poland
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Unverified
Germany

Common questions

What is primary immunodeficiency?

It's a condition where your body's immune system, which normally fights off germs, doesn't work as well as it should, leading to more frequent or severe infections.

What is Newnorm?

Newnorm is a new medicine of human immunoglobulin, given as an injection under the skin, designed to help boost your body's immune defences.

How long will the study last?

The main part of the study where we check how well the medicine works will last for 52 weeks, which is about one year.

Will I know if I'm getting Newnorm?

Yes, this is an 'open-label' study, meaning both you and the doctors will know you are receiving Newnorm.

What kind of infections will the study be looking at?

The study will focus on serious bacterial infections like blood poisoning or severe pneumonia, but also other common infections, and their impact on your life.