Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

This research study, called PRESent-2, is testing a new medication called SerpinPC. It's for adults with severe haemophilia A or moderately severe to severe haemophilia B. The main goal is to find out if SerpinPC can help reduce the number of bleeding episodes these patients experience, especially those who currently only treat bleeds when they occur (this is called 'on-demand' treatment). Researchers will carefully compare bleeding rates before and after taking SerpinPC. They will also be looking closely at how safe the new medicine is and how it affects people's quality of life. This is a Phase 2 study, meaning it's exploring how well the medicine works and its safety in a smaller group of people.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Apcintex Limited

Enrolment target

Start

25 Jul 2023

Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

What is this study about?

This research study is looking into a new medicine called SerpinPC for people who have severe haemophilia A or moderately severe to severe haemophilia B. Haemophilia is a condition where your blood doesn't clot properly, which can lead to frequent bleeding, often into joints and muscles. This study wants to see if SerpinPC can make a real difference in reducing these bleeding episodes.

The main thing researchers want to find out is whether SerpinPC can lower how often people who currently treat bleeds as they happen (sometimes called 'on-demand' treatment) actually experience them. They will compare the number of bleeds a person has before starting SerpinPC with the number of bleeds they have while taking the medicine. They're also interested in how safe SerpinPC is and whether it affects people's overall quality of life.

This is an important step in finding new ways to help people living with haemophilia. If SerpinPC proves to be effective and safe, it could offer a new treatment option for managing bleeding and improving daily life for those with the condition.

Key takeaways

This study is testing a new medicine called SerpinPC for people with severe haemophilia A or B.
The main goal is to see if SerpinPC can reduce bleeding episodes.
It's a Phase 2 study, focusing on how well the medicine works and its safety.
Participants will have regular clinic visits, blood tests, and health checks.
You can stop participating at any time.
The study is for adult men aged 18 and over.

Who may be eligible?

To take part in this study, you need to be an adult, aged 18 or older. The study is specifically for men.

You must have been diagnosed with severe haemophilia A or moderately severe to severe haemophilia B. These are specific types of haemophilia that the study is focusing on.

There might be other health requirements or medications you are taking that would mean you couldn't join, but these would be discussed with you in detail by the study team.

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?
Are you a man?
Do you have severe haemophilia A?
Or do you have moderately severe to severe haemophilia B?
Are you currently treating bleeds only when they happen ('on-demand')?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first have a screening visit to make sure it's suitable for you. After that, there will be an observation period where your usual medical care and bleeding episodes would be recorded. Then, you would start taking the study medicine, SerpinPC, regularly for a period.

The study involves regular visits to the clinic where you would have blood tests, physical examinations, and answer questions about your health and any bleeding episodes. You would also track any bleeds and possibly fill out questionnaires about how you are feeling and your quality of life. The study plans to look into your health up to 24 weeks after you start taking SerpinPC. The total duration of your involvement could be longer, depending on how the study progresses, but you would be given full details of the schedule.

Potential risks and benefits

Taking part in a clinical study means you might get access to a new treatment before it's widely available, which could potentially reduce your bleeding episodes if SerpinPC works well for you. However, there's no guarantee the treatment will be effective, and you might not experience any direct benefit. All medicines have potential side effects, and SerpinPC could have risks that are not yet fully known. The study team will closely monitor your health to identify any issues quickly. Remember, joining is entirely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (7)

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
Germany
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Unverified
Belgium
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Unverified
Italy
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Unverified
France
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Unverified
Poland
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Unverified
Bulgaria

Common questions

What is haemophilia?

Haemophilia is a genetic condition where your blood doesn't clot properly, leading to longer bleeding and easy bruising.

What is SerpinPC?

SerpinPC is the name of the investigational medicine being tested in this study for treating haemophilia.

What does 'on-demand' treatment mean?

On-demand treatment means you only take medicine to stop bleeding once it has already started, rather than regularly to prevent it.

Will I know if I'm getting SerpinPC?

This is an 'open-label' study, which means both you and your study doctor will know that you are receiving SerpinPC.

How long will the study last for me?

The main part of the study where your bleeds are assessed will last up to 24 weeks after you start SerpinPC, but your total involvement could be longer.