Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Hemophilia B With Inhibitors (PRESent-3)

This study, called PRESent-3, is for adult men living with Haemophilia B who have developed a complication called 'inhibitors'. These inhibitors can make it harder for their usual treatments to work. We are testing a new medicine called SerpinPC. The main goal is to see how well SerpinPC reduces the number of bleeding episodes these men experience over a certain period. We will also be carefully monitoring for any side effects to make sure SerpinPC is safe. This is a Phase II study, meaning we're exploring if this new treatment works and if it should be studied further. The results will help us understand if SerpinPC could become a helpful new option for people with this condition.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Apcintex Limited

Enrolment target

Start

07 Aug 2023

Hemophilia B

What is this study about?

This study is about a condition called Haemophilia B, which is a rare blood disorder where the blood doesn't clot properly. People with Haemophilia B can experience bleeding episodes, which need treatment. Some men with Haemophilia B develop something called 'inhibitors'. These inhibitors are like antibodies that attack the usual medicines designed to help their blood clot, making those treatments less effective. This can make managing bleeding much more difficult.

Researchers are investigating a new medicine called SerpinPC. The main aim of this study is to find out if SerpinPC can help reduce the number of treated bleeds (times when bleeding happens and needs medical attention) in men with Haemophilia B and inhibitors. We will compare the number of bleeds before starting SerpinPC to the number of bleeds after taking the medicine for a period. We'll also be closely watching for any side effects to understand how safe SerpinPC is.

This study is in an exploratory phase (Phase II), which means it's one of the earlier stages of drug development. We are trying to determine if SerpinPC works as expected and if it's safe enough to continue studying in larger groups of people. The information gathered from this study is really important for potentially developing new and better treatment options for people living with Haemophilia B and inhibitors.

Key takeaways

This study is for adult men with Haemophilia B and inhibitors.
It's testing a new medicine called SerpinPC to reduce bleeding episodes.
Doctors will monitor your bleeds and check for any side effects.
Participation involves regular clinic visits and providing blood samples.
This is an early-stage study to understand if SerpinPC is effective and safe.

Who may be eligible?

This study is specifically designed for men who are 18 years old or older. The key requirement is that you must have a confirmed diagnosis of Haemophilia B.

Crucially, you must also have developed 'inhibitors.' These are special antibodies that make your body respond differently to the usual treatments for Haemophilia B. If you're not sure if you have inhibitors, your doctor will be able to confirm this.

There will be other detailed medical checks to make sure this study is safe and right for you, but these are the main points. If you meet these general criteria, you might be suitable for this study.

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 male?
Are you 18 years old or older?
Do you have Haemophilia B?
Has your doctor told you that you have developed inhibitors?

Answer every question to see your result.

What does participation involve?

If you join this study, you will first have an 'observation period' where your bleeds are monitored before you start SerpinPC. Then, you will receive the study medicine, SerpinPC, for a period. This will involve regular visits to the clinic for check-ups, where doctors will take blood samples, ask about your health, and record any bleeding episodes you experience. We will also look at how SerpinPC levels are in your blood. You may also be asked to complete questionnaires about your quality of life, especially if you are between 17 and 65 years old. The total time you would be involved in the study would depend on how long you receive the treatment, but it will involve careful monitoring for at least 24 weeks once you start SerpinPC, and potentially up to 48 weeks. You can withdraw from the study at any time.

Potential risks and benefits

Taking part in a clinical trial has both potential benefits and risks. The potential benefit is that SerpinPC might help reduce your bleeding episodes, potentially offering a new treatment option for your Haemophilia B with inhibitors. However, as SerpinPC is a new medicine, there's a chance it might not work as hoped, or you could experience side effects that we don't fully know about yet. All known and potential risks will be thoroughly explained to you before you decide to join. It's important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

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

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Unverified
Spain
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Unverified
France
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Unverified
Italy
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Unverified
Germany

Common questions

What is Haemophilia B?

Haemophilia B is a health condition where your blood doesn't clot properly, leading to bleeding that can be hard to stop.

What are 'inhibitors' in Haemophilia B?

Inhibitors are like antibodies your body can make that stop your usual haemophilia medicine from working as well as it should.

What is SerpinPC?

SerpinPC is a new medicine being tested to see if it can help reduce bleeding in men with Haemophilia B who have inhibitors.

What does 'Phase II' mean for a study?

Phase II means it's an earlier stage of testing where we're looking to see if the medicine works and if it's safe enough to study further.

Will I have to pay to be part of the study?

No, you will not have to pay to participate in the study. All study-related treatments and tests will be provided free of charge.