Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Open-label extension study with Tadekinig alfa (r-hIL-18BP) to monitor safety and tolerability in patients with IL-18 driven monogenic autoinflammatory conditions: NLRC4 mutation and XIAP deficiency

This research is an ongoing study for a medicine called Tadekinig alfa. It's for people who have certain rare conditions where their body's immune system mistakenly causes inflammation. These conditions are linked to specific genetic changes like NLRC4 mutation and XIAP deficiency, which involve a molecule called IL-18. The main purpose of this study is to keep a close eye on how safe the medicine is and if people experience any side effects. We'll also check if the body's immune system reacts to the medicine and how the medicine works in the body. This is a follow-up to earlier studies, aiming to confirm its benefits and safety over a longer period.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

AB2 Bio S.A.

Enrolment target

Start

04 Jul 2023

18 driven monogenic autoinflammatory conditions: NLRC4 mutation and XIAP deficiency

What is this study about?

This study is an ongoing project looking into a medicine called Tadekinig alfa. It's designed for people who have specific, rare conditions where their body's natural defence system, or immune system, mistakenly causes too much inflammation. These conditions are often present from birth and are due to changes in certain genes, specifically something called an NLRC4 mutation or XIAP deficiency.

In these conditions, a molecule in the body called IL-18 becomes overactive, leading to constant inflammation. Tadekinig alfa is being tested to see if it can help by blocking the effects of this overactive IL-18. This study is an 'open-label extension', which means that participants and their doctors will know they are receiving the study medicine, and it's a continuation for people who may have already taken part in an earlier study of Tadekinig alfa. The main goal is to carefully watch how safe the medicine is over a longer period and see how well people tolerate it.

Understanding the safety and how patients manage the treatment is extremely important for new medicines. This study will help doctors learn more about any potential side effects and how the medicine affects the body over time for these specific, rare inflammatory conditions. It's a crucial step in potentially making this treatment available to more people in the future.

Key takeaways

This study explores the long-term safety of Tadekinig alfa for rare inflammatory conditions.
It's for people with NLRC4 mutation or XIAP deficiency.
Participants will have regular check-ups to monitor health and side effects.
It's a continuation for those who have previously taken Tadekinig alfa.
Your participation helps advance knowledge for these conditions.

Who may be eligible?

This study is open to both adults and children, male and female, who have already taken part in a previous study involving Tadekinig alfa.

To be eligible, participants must have one of two specific rare inflammatory conditions linked to an overactive molecule called IL-18. These conditions are known as NLRC4 mutation-associated autoinflammatory disease or XIAP deficiency. If you have been diagnosed with one of these conditions and have previously received Tadekinig alfa in another study, you might be able to join this one.

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 have been diagnosed with an NLRC4 mutation or XIAP deficiency.
I have previously participated in a study involving Tadekinig alfa.
I am comfortable with regular medical check-ups and blood tests.
I am willing to follow the study's treatment plan.
I understand I can withdraw from the study at any time.

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be given the study medicine, Tadekinig alfa. Throughout the study, you'll have regular visits with the study team. During these visits, the doctors and nurses will monitor your health by asking about any changes you've noticed, performing physical examinations, and taking blood and urine samples. These tests help them check for any side effects and see how your body is reacting to the medication. They will also assess how your body handles the medicine and how it works to control inflammation.

The study is designed to follow you over a longer period, acting as a continuation of previous treatments. The exact number of visits and the full duration of your participation will be explained in detail by the study team, as it can vary slightly depending on your individual situation and your doctors' assessment.

Potential risks and benefits

A potential benefit of joining this study is the chance to continue receiving a treatment that may help manage your inflammatory condition, especially if you have previously responded well to Tadekinig alfa. You would also be contributing valuable information that could help others with similar conditions in the future. However, like all medicines, Tadekinig alfa might have side effects, which will be carefully monitored throughout the study. These could include reactions at the injection site or other effects on your body. You are entirely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Germany

Common questions

What kind of conditions is this study for?

This study is for people with rare inflammatory conditions caused by specific genetic changes, like NLRC4 mutation or XIAP deficiency, where a molecule called IL-18 causes too much inflammation.

What is Tadekinig alfa?

Tadekinig alfa is the medicine being tested. It's designed to help block the effects of the overactive IL-18 molecule, aiming to reduce inflammation in these conditions.

Why is this study being done?

This study is a follow-up to previous research. Its main goal is to monitor the long-term safety of Tadekinig alfa and see how well people tolerate it when used over time.

Who can join this study?

This study is for both children and adults, male or female, who have one of the specific conditions and have already taken part in an earlier study of Tadekinig alfa.

Will I know if I am getting the real medicine?

Yes, this is an 'open-label' study, meaning both you and your doctor will know that you are receiving Tadekinig alfa.