Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

(19429) A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A

This clinical trial is looking at a new treatment called BAY 2599023 for adult men who have severe haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly. The treatment is a type of gene therapy, which means it tries to correct the problem by giving your body instructions to make the missing clotting factor (called Factor VIII). This study is in its early stages (Phase 1 and 2), meaning it's one of the first times it's being used in people. Researchers want to understand how safe it is, what side effects might happen, and what the best dose is. They will also check if it helps the body produce more Factor VIII.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Bayer AG

Enrolment target

Start

18 Dec 2023

Hemophilia A

What is this study about?

This study is about a new and promising approach to treating severe haemophilia A in adult men. Haemophilia A is a genetic condition where the body doesn't produce enough of a protein called Factor VIII, which is essential for blood clotting. This means people with severe haemophilia A can experience bleeding episodes that are hard to stop.

The new treatment being tested is called BAY 2599023. It's a type of 'gene therapy'. Imagine your body's cells are like tiny factories. In haemophilia A, the factory doesn't have the right instructions to make Factor VIII. This gene therapy aims to deliver new, correct instructions to these factories using a harmless 'delivery vehicle' (called an AAV) to help your body start making its own Factor VIII again. This could potentially reduce or even stop bleeding episodes.

Because this is a new treatment, the study is in its early stages (Phase 1 and 2). This means researchers are focused on making sure the treatment is safe for people to take and finding the best dose. They will be carefully monitoring participants for any side effects and seeing how well their bodies start producing Factor VIII after the treatment. This is an important step towards finding better ways to manage haemophilia A.

Key takeaways

This is an early-stage study (Phase 1/2) for a new gene therapy for severe haemophilia A.
It aims to find a safe dose and see how well it helps the body produce Factor VIII.
Only adult men with severe haemophilia A can participate.
Participation involves a single treatment and regular check-ups.
Potential benefits include better clotting, but risks are not yet fully understood.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you would need to be an adult man, aged 18 years or older, who has been diagnosed with severe haemophilia A. Severe haemophilia A means your body produces very little or no Factor VIII, which is the protein needed for your blood to clot properly.

The study is specifically looking for people who can safely receive this new gene therapy. There will be other detailed medical checks to make sure your overall health is suitable for participating in a clinical trial like this. These checks would ensure that the treatment has the best chance of working safely for you and that you don't have other health conditions that might make the treatment too risky.

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 years or older?
Are you male?
Have you been diagnosed with severe haemophilia A?
Are you willing to attend regular follow-up appointments?
Are you generally in good health apart from your haemophilia A?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive the study treatment, BAY 2599023, which is given as a single infusion into your vein. After receiving the treatment, you would have regular hospital visits for medical check-ups, blood tests, and to make sure you are feeling well. These visits are very important for the research team to monitor your health and see how your body is responding to the treatment. The total duration of your participation in this study, including the treatment and follow-up, would be carefully explained to you, but typically early phase gene therapy studies involve long-term follow-up to ensure safety and effectiveness.

Potential risks and benefits

Participating in this study might offer the potential benefit of improving your haemophilia A by helping your body produce more Factor VIII, which could lead to fewer bleeding episodes. However, as with any new treatment, there are potential risks, including side effects from the treatment or from the procedures and tests involved. The exact side effects are not fully known in humans yet, as this is an early-stage study. All potential benefits and risks will be discussed with you in detail, and you have the right to withdraw from the study at any time, for any reason, without it affecting your 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
Netherlands
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Unverified
Germany
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Unverified
France
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Unverified
Bulgaria

Common questions

What is gene therapy?

Gene therapy aims to fix a problem by giving your body new instructions (genes) to correct a faulty one, like helping your body make a missing clotting factor for haemophilia.

Is this a cure for haemophilia A?

This study is testing a new treatment and is in early stages. It aims to help your body produce Factor VIII, which could reduce bleeding, but it is not yet known if it will be a cure.

What is Factor VIII?

Factor VIII is a protein in your blood that helps it clot properly. People with haemophilia A don't have enough of it, leading to bleeding problems.

How will the treatment be given?

The treatment, BAY 2599023, will be given as a single dose into a vein, like a drip.

Will I know my results?

As part of your participation, the study team will discuss with you how your health and Factor VIII levels are changing throughout the study.