Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult aHUS patients who are naive to complement inhibitor therapy

This study is testing a new medicine, IPTACOPAN, for adults with a rare and serious kidney condition called atypical hemolytic uremic syndrome (aHUS). This condition causes blood problems and can damage the kidneys. The study is particularly for patients who haven't tried similar medicines before. Researchers want to find out if IPTACOPAN can effectively help blood and kidney functions get better over 26 weeks, reducing the need for treatments like plasma exchange or anti-C5 antibody therapies. They will check if blood counts improve, if kidney function gets better, and if patients feel better overall. The study also aims to understand any side effects of the medicine. It's a Phase III trial, meaning it's a later stage of testing, aiming to confirm the medicine's effectiveness and safety.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Novartis Pharma AG

Enrolment target

Start

01 Jul 2024

Atypical hemolytic uremic syndrome

What is this study about?

This study is about a new medicine called IPTACOPAN, which is being tested for adult patients who have a rare and serious kidney disease known as atypical hemolytic uremic syndrome (aHUS). This condition can cause serious problems with your blood and damage your kidneys. The study is especially for people who haven't yet used a specific type of medicine called a 'complement inhibitor' for their aHUS.

The main goal of the study is to see if IPTACOPAN can help improve your blood counts and kidney function over a period of 26 weeks (about six months). The researchers are looking to see if your blood (platelet count and an enzyme called LDH) returns to normal and if your kidney function gets significantly better. This improvement should happen without needing some other common treatments for aHUS, like plasma exchange or anti-C5 antibody therapies. They will also keep a close eye on how you're feeling and whether your quality of life improves during the study.

Beyond checking these main improvements, the study will also look at other things. This includes how quickly any improvements in your blood and kidney function happen, whether you need dialysis less often, and how your general health and tiredness change. Most importantly, the study will carefully check for any side effects of the new medicine to make sure it is safe.

Key takeaways

This study is for adults with atypical hemolytic uremic syndrome (aHUS).
It's testing a new medicine called IPTACOPAN taken twice daily.
The main aim is to see if IPTACOPAN improves blood and kidney problems.
Participants should not have previously received certain aHUS treatments.
The study lasts for 26 weeks, with regular health checks.
Researchers will also examine how the medicine affects your quality of life and any side effects.

Who may be eligible?

This study is looking for adult patients, meaning you must be at least 18 years old.

To be considered for this study, you must have been diagnosed with atypical hemolytic uremic syndrome (aHUS). An important part of the eligibility is that you should not have received 'complement inhibitor' therapy for your aHUS before. This means the study is focused on patients who are new to this particular type of treatment.

The study team will carefully review other health information to make sure it's safe and appropriate for you to participate. This helps ensure the study results are clear and that participants are well cared for.

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?
Have you been diagnosed with atypical hemolytic uremic syndrome (aHUS)?
Have you NOT received a specific type of aHUS treatment called a 'complement inhibitor' before?
Are you able to take a study medicine by mouth twice a day?

Answer every question to see your result.

What does participation involve?

If you join this study, you would take a new medicine called IPTACOPAN by mouth, twice a day. The study treatment period lasts for 26 weeks, which is about six months. During this time, you would have regular visits to the clinic so the study team can check your health, take blood and urine samples, and monitor your kidney function. They will also ask you how you are feeling and if you have any new symptoms or side effects.

You will complete questionnaires about your quality of life and how tired you feel. The doctors will closely track your platelet count, kidney function, and other important health markers. The total duration of your involvement, including follow-up, will be determined by the study protocol, but the main treatment period is 26 weeks.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of this study is that you might receive a new medicine, IPTACOPAN, which could help improve your aHUS symptoms, blood problems, and kidney function, possibly reducing the need for other treatments. However, there's no guarantee the medicine will work for you, and it could have side effects, some of which might be serious. The study team will explain all known side effects and monitor you very 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
Slovenia
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Unverified
Slovakia
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Unverified
Czechia
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Unverified
Greece
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Unverified
Austria

Common questions

What is aHUS?

Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes tiny blood clots to form in your small blood vessels, which can damage organs, especially the kidneys.

What is IPTACOPAN?

IPTACOPAN is a new medicine being tested in this study to see if it can help treat aHUS by targeting certain parts of your immune system.

How long will I take the study medicine?

If you join the study, you would take the study medicine daily for 26 weeks, which is about six months.

Will I still get my usual treatment for aHUS?

This study is for patients who haven't had certain aHUS treatments before, and it aims to see if IPTACOPAN can be effective without needing other specific therapies like plasma exchange or anti-C5 antibodies.

Are there any side effects?

Like all medicines, IPTACOPAN may have side effects. The study team will explain all known side effects before you decide to join and will carefully monitor you for any during the study.