Not recruitingRare disease

Study on the Safety and Effects of BAY 2599023 (Peboctocogene Camaparvovec) for Adults with Severe Hemophilia A

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Hemophilia A, which is a genetic disorder that affects the blood's ability to clot properly. The study is testing a new treatment called BAY 2599023 (also known as DTX201). This treatment uses a modified, non-infectious virus to deliver a healthy version of the defective Factor VIII gene into the liver cells. The goal is to help the body produce the necessary protein for blood clotting.

The purpose of the study is to investigate the safety and how well patients with severe Hemophilia A respond to this new treatment. Participants will receive a single dose of the treatment through an intravenous (IV) infusion. The study will monitor the participants over time to see how their bodies react to the treatment and to check for any side effects. The study will also measure how much of the Factor VIII protein is produced in the body after receiving the treatment.

Throughout the study, researchers will keep track of any adverse events, which are any unwanted effects that might occur. They will also look at how well the treatment works by measuring the levels of Factor VIII in the blood at different times. The study aims to find out if the treatment can help increase the levels of Factor VIII to a point where it can effectively help with blood clotting in patients with severe Hemophilia A.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit
Upon joining the study, you will attend an initial visit. During this visit, your eligibility will be confirmed based on specific criteria, such as age and medical history related to hemophilia A.
You will be informed about the study procedures, and your consent will be obtained to participate in the trial.
Step 2
Treatment administration
You will receive a single dose of the study medication, BAY 2599023, which is administered through an intravenous (IV) infusion. This means the medication is delivered directly into your bloodstream through a vein.
The medication is a gene therapy designed to deliver a healthy version of the defective Factor VIII gene into your liver cells using a modified, non-infectious virus.
Step 3
Monitoring and follow-up
After receiving the treatment, you will be monitored closely for any side effects or adverse reactions. This includes regular check-ups and tests to assess your health and the effectiveness of the treatment.
You will be required to attend follow-up visits at specified intervals to evaluate the expression of Factor VIII activity in your body. These visits will help determine how well the treatment is working over time.
Step 4
Long-term follow-up
The study includes a long-term follow-up period to continue monitoring your health and the treatment's effects. This period may last up to several years, with visits scheduled at regular intervals.
During this time, you will be asked to provide blood samples and other necessary information to help researchers understand the long-term safety and effectiveness of the treatment.

Who can join the trial?

8 criteria

Must be a male who is 18 years or older.
Must have severe hemophilia A, which means having very low levels of a blood clotting protein called FVIII (less than 1%).
Must have been treated with FVIII products, which are either made from plasma or created in a lab, for at least 150 days.
Must be on one of these treatments:
- Prophylaxis: Regular treatment to prevent bleeding, and willing to stop this treatment at certain times during the study.
- On-demand: Treatment only when bleeding occurs, and must have had more than 4 bleeding events in the past year.
- Must agree to use double barrier contraception methods, such as condoms with a spermicidal agent, from the time of receiving the study drug until told otherwise by the study doctor. If the partner uses an intra-uterine device or hormone-based contraception, an additional barrier method must be used.
- If vasectomized, must still agree to use condoms.
- Must agree not to donate cells, semen, blood, tissue, or organs from the time of receiving the study drug.

Who cannot join the trial?

3 criteria

Patients who do not have severe hemophilia A cannot participate. Hemophilia A is a condition where blood does not clot properly due to a lack of a specific protein.
Only adult male patients can participate. This means female patients and children are not eligible.
Patients who have not been previously treated with FVIII product cannot participate. FVIII is a treatment used to help blood clot in people with hemophilia A.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Amsterdam UMC
Amsterdam, The Netherlands
Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands
Verified
Goethe University Frankfurt
Frankfurt, Germany

Trial locations

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Investigated drugs

BAY 2599023 (DTX201) is a type of gene therapy being studied for its potential to help people with severe hemophilia A. This therapy uses a special virus, called an adeno-associated virus, to deliver a modified version of a human protein known as factor VIII into the body. People with hemophilia A have low levels of factor VIII, which is important for blood clotting. By introducing this modified factor VIII, the therapy aims to help the body produce more of this protein on its own, potentially reducing bleeding episodes and improving quality of life for patients. The study is focused on understanding how safe and tolerable this therapy is when given in different amounts to adults who have already been treated with other factor VIII products.

Peboctocogene Camaparvovec

What is already known about the treatment

BAY 2599023 (DTX201) – This medication is administered through a single intravenous (IV) infusion, meaning it is delivered directly into the bloodstream. It is currently being studied in clinical trials to assess its safety and effectiveness for treating severe Hemophilia A, a condition where blood does not clot properly. The medication works by using a modified virus to deliver a gene that helps produce a protein called factor VIII, which is essential for blood clotting. It falls under the category of gene therapy, which aims to treat diseases by correcting genetic defects.

Investigated diseases

Hemophilia A – Hemophilia A is a genetic disorder caused by a deficiency of clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Individuals with Hemophilia A may experience frequent nosebleeds, easy bruising, and bleeding into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to joint damage and reduced mobility. The severity of the disease varies, with some individuals experiencing mild symptoms and others having severe bleeding episodes. Hemophilia A is typically inherited in an X-linked recessive pattern, affecting mostly males.