Ongoing, recruitingHuman Pharmacology (Phase I)- Bioequivalence StudyInterventional

Pharmacokinetic comparison of efanesoctocog alfa vs other EHL-rFVIII products in participants with severe haemophilia A

This research study is for men with severe haemophilia A. It's looking at a new medicine called efanesoctocog alfa. The main goal is to compare how this new medicine behaves in the body – how quickly it's absorbed, how long it lasts, and how it's cleared – compared to other similar treatments already available. This type of study, called a Phase I or 'Human Pharmacology' study, is usually one of the first steps in testing a new medicine in people. By understanding how the medicine works inside the body, researchers can learn if it's acting as expected and if it could be a good potential treatment option for haemophilia A.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Bioequivalence Study

Sponsor

Swedish Orphan Biovitrum AB (publ)

Enrolment target

Start

03 Apr 2024

Haemophilia A

What is this study about?

This study is about a new medicine called efanesoctocog alfa, which is being developed to help people with severe haemophilia A. Haemophilia A is a condition where a vital part of the blood clotting system, a protein called Factor VIII, is either missing or doesn't work properly. This means people with haemophilia A can have bleeding problems.

The main idea of this study is to compare how this new medicine (efanesoctocog alfa) works in the human body compared to other treatments that are already used for haemophilia A. Scientists call this 'pharmacokinetics'. It means they want to see how the body handles the medicine – how it's taken in, how it travels around, how long it stays active, and how it leaves the body. Understanding this helps researchers find out if the new medicine is as effective, or potentially even more effective and convenient, than current treatments.

This specific study is a 'Phase I' trial. This is an early stage of testing new medicines in people. In this phase, the main goal is often to see if the medicine is safe and how it acts in the body, rather than how well it treats the condition itself. The information gathered from this study will be very important for deciding if efanesoctocog alfa should be tested further in larger studies, hopefully leading to a new treatment option for people with severe haemophilia A.

Key takeaways

This study is testing a new medicine, efanesoctocog alfa, for severe haemophilia A.
It compares how this new medicine acts in the body versus existing treatments.
It's an early-stage study (Phase I) focused on safety and how the medicine works.
Only adult men (18+) with severe haemophilia A can participate.
Participation involves clinic visits, blood tests, and receiving study medication.
You have the right to leave the study at any time.

Who may be eligible?

This study is specifically looking for people who have severe haemophilia A. You would need to be diagnosed with this condition to be considered for the study.

Also, to take part, you must be an adult – aged 18 years or older. There is no upper age limit, meaning older adults can also participate if they meet all other requirements.

This study is open only to men. This is because haemophilia A almost exclusively affects males. If you are a woman or a younger person under 18, unfortunately, you would not be able to join this particular 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.

Do I have a diagnosis of severe haemophilia A?
Am I 18 years old or older?
Am I male?
Am I able to attend regular clinic appointments and follow study rules?
Am I currently on any other treatments for haemophilia that might affect participation?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the study medication, efanesoctocog alfa, and potentially other similar treatments for comparison. You would have regular visits to the study clinic or hospital. During these visits, doctors and nurses would perform various assessments. This might include taking blood samples to check how the medicine is working in your body and monitoring your general health. You would also be asked about any side effects you might experience.

The specific number of visits and the total length of your participation would be explained fully by the study team. They will make sure you understand the schedule, including any follow-up appointments after you stop taking the study medication. It's important to commit to attending all scheduled appointments to help the researchers gather complete information.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of joining this study could be access to a new treatment for severe haemophilia A that isn't yet widely available. Your participation also helps researchers learn more about haemophilia A, which could lead to better treatments for everyone in the future. However, there's no guarantee the new medicine will help you personally. Potential risks could include side effects from the study medication, which might be mild or, in rare cases, more serious. There might also be discomfort from blood draws or other study procedures. Your doctors will carefully explain all known risks before you decide to join. Remember, taking part is completely voluntary, and you have the right to withdraw from 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
Germany
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Unverified
Spain
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Unverified
Italy
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Unverified
Bulgaria

Common questions

What is haemophilia A?

Haemophilia A is a genetic bleeding disorder where the blood doesn't clot properly because of a problem with a protein called Factor VIII.

What is a 'Phase I' study?

A Phase I study is an early step in testing a new medicine in people. It mainly checks if the medicine is safe and how the body handles it.

What does 'pharmacokinetic comparison' mean?

It means the study is comparing how a new medicine moves through and acts in the body compared to other similar medicines.

Will I get the new medicine?

If you join, you would receive the new medicine or another similar treatment, depending on what the study design involves.

Who is running this study?

This study is organised by researchers and doctors who specialise in haemophilia and new medicines.