Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A study to test a medicine (fitusiran) for preventing bleeds in people with severe hemophilia who previously received preventive treatment with emicizumab

This research study is looking into a new medication called fitusiran. It's designed for adult men who have severe haemophilia A, a condition where your blood doesn’t clot properly, leading to bleeding. Currently, these men are already using another preventative medicine, emicizumab, but are still experiencing bleeding episodes. The main goal of this study is to find out if fitusiran can be effective in preventing bleeds for these individuals. This is an early-stage study, meaning researchers are cautiously exploring how the medicine works in people. It will help us understand if fitusiran could be a helpful new option for managing severe haemophilia A.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Sanofi-Aventis Research & Development

Enrolment target

Start

05 Feb 2024

Hemophilia A

What is this study about?

This study is about an important condition called haemophilia A. People with haemophilia A have a problem with their blood clotting, which can lead to bleeding that is hard to stop. Most people with severe haemophilia A take medicine regularly to try and prevent these bleeds. This is often called 'preventative treatment' or 'prophylaxis'.

Sometimes, even with preventative medicine, people still experience bleeding. The medicine being tested, fitusiran, is a new type of treatment. This study is specifically looking at adult men who have severe haemophilia A and are already taking another preventative medicine called emicizumab, but are still having bleeding problems. The aim is to see if fitusiran can offer an additional or alternative way to help prevent these bleeds.

This is an early stage of human testing for fitusiran, sometimes called a 'Phase I' study. This means the researchers are carefully observing how the medicine works in the body, checking for safety, and seeing what doses might be effective. The information gathered from this study will be crucial for understanding if fitusiran has the potential to become a useful new treatment option for managing severe haemophilia A.

Key takeaways

Tests a new medicine, fitusiran, for severe haemophilia A.
Aims to prevent bleeds in adult men still bleeding despite emicizumab.
Early stage (Phase I) study checking safety and how the medicine works.
Participation involves regular clinic visits and close monitoring.
Potential for reduced bleeds, but risks include unknown side effects.

Who may be eligible?

To be considered for this study, you would need to be an adult man, 18 years old or older. The study is specifically looking for men who have been diagnosed with severe haemophilia A.

It's also important that you have been receiving preventative treatment with another medicine called emicizumab before joining this study. This study is for people who, despite taking emicizumab, are still experiencing bleeding episodes.

There may be other specific health requirements or conditions that would determine if this study is suitable for you. Your doctor or the study team would go through all these details to see if you meet the full criteria.

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 an adult aged 18 or over?
Are you a man?
Do you have a diagnosis of severe haemophilia A?
Are you currently receiving preventative treatment with emicizumab?
Are you still experiencing bleeding episodes despite taking emicizumab?

Answer every question to see your result.

What does participation involve?

As this is an early-stage study, taking part would involve several visits to the study clinic. You would likely have regular health checks, blood tests, and other assessments to monitor your health and how the new medicine is working. You would be given the study medicine, fitusiran, according to a schedule set by the researchers.

Throughout your time in the study, doctors and nurses will closely monitor you. They will check for any side effects and see how your body responds to fitusiran. You would also need to keep track of any bleeding events you experience. The total length of your participation would be explained fully by the study team, but early-stage studies often involve close monitoring over several weeks or months.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study could be that fitusiran might help reduce your bleeding episodes if your current treatment isn't fully effective. You would also be contributing to medical knowledge that could help future patients. However, there are potential risks, including side effects from the new medicine, which are not yet fully known. There is also no guarantee that the medicine will help your condition. You would be fully informed about all known risks before deciding to join, and you have the right to withdraw from the study at any time without affecting your usual medical care.

Locations (2)

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
Spain

Common questions

What is haemophilia A?

Haemophilia A is a inherited condition where your blood doesn’t clot properly, which can lead to longer bleeding after an injury or even spontaneous bleeds.

What is fitusiran?

Fitusiran is a new medicine being tested. It aims to help people with severe haemophilia A to prevent bleeding episodes.

Why is this study only for men?

Haemophilia A is a genetic condition that almost exclusively affects men. Women are typically carriers of the condition but usually don't experience symptoms.

What does 'preventative treatment' mean?

Preventative treatment means taking medicine regularly to try and stop bleeding episodes from happening, rather than treating them once they start.

Will I still take my usual haemophilia medicine?

The study is for people who are already on emicizumab. The study team will explain exactly how fitusiran would be given, and whether it's in addition to or instead of your current medication for the study duration.