Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
This research is looking at a new treatment called gene therapy, specifically BAY 2599023 (DTX201), for adult men who have severe haemophilia A. Haemophilia A is a condition where your blood doesn't clot properly because you don't have enough of a protein called Factor VIII. The new treatment works by delivering a healthy version of the Factor VIII gene into your liver cells using a specially modified, harmless virus. The hope is that this will allow your body to start making its own Factor VIII, which could reduce bleeding incidents and the need for regular treatments. The study aims to find out if this treatment is safe and how well it works, as well as finding the best dose.
At a glance
What is this study about?
Imagine your body is like a finely tuned machine, and a crucial part of that machine helps your blood to clot when you get a cut or bruise. If you have haemophilia A, it means this clotting part, a protein called Factor VIII, isn't working as it should, or you don't have enough of it. This can lead to unexpected bleeds and other health problems. Currently, many people with haemophilia A need regular treatments to replace this missing Factor VIII.
This study is exploring a new and innovative way to tackle haemophilia A, called gene therapy. Instead of constantly topping up the missing Factor VIII from outside, this treatment, called BAY 2599023 (DTX201), tries to teach your body to make its own. It does this by using a harmless, specially altered virus, like a tiny delivery driver, to carry a healthy copy of the Factor VIII gene into your liver cells. Once in your liver, the healthy gene should be able to instruct your cells to produce the Factor VIII protein you're missing.
The main goal of this particular study is to understand if BAY 2599023 (DTX201) is safe for people with severe haemophilia A, and how effective it is at helping their bodies produce Factor VIII. Researchers are also trying to find the optimal amount of the treatment to give to get the best results. If successful, this kind of gene therapy could potentially offer a long-lasting solution, reducing the burden of current treatments and improving quality of life for those with haemophilia A.
Key takeaways
- This study is testing a new gene therapy for severe haemophilia A.
- It aims to help your body produce its own clotting Factor VIII.
- The treatment is given as a single infusion.
- It's only for adult men (18+) with severe haemophilia A.
- You will be closely monitored for safety and effectiveness for a long time.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is specifically looking for adult men, aged 18 or over, who have been diagnosed with severe haemophilia A. To confirm this, doctors will check your medical history and blood tests to make sure your Factor VIII levels are very low (less than 1% of normal).
You would generally need to have had a fair amount of experience with previous Factor VIII treatments. If you usually take preventative treatments, you’d need to be prepared to stop these at certain times during the study. If you only take treatment when you have a bleed, you should have had more than four bleeding episodes in the past year.
There are also some reasons why you might not be able to join. For example, if you've had allergic reactions to Factor VIII treatments before, have certain other health conditions like severe liver disease, uncontrolled HIV, or severe obesity, or have specific antibodies in your blood that would stop the treatment from working. You also cannot be currently pregnant or trying to get pregnant, and you must agree to use reliable contraception during the study. You also can't have participated in another gene therapy trial recently.
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 male (18 years or older)?
- Do you have a confirmed diagnosis of severe haemophilia A (Factor VIII less than 1%)?
- Have you had previous Factor VIII treatments (over 150 times)?
- If you have 'on-demand' treatment, have you had more than 4 bleeds in the last year?
- Are you willing and able to use reliable contraception?
- Do you NOT have serious other health conditions like severe liver disease or uncontrolled HIV?
What does participation involve?
If you decide to take part in this study, you will receive the study drug, BAY 2599023 (DTX201), which is given as a single infusion (into a vein). Before and after this treatment, you will have regular hospital visits for check-ups and tests. These tests will include blood samples to measure your Factor VIII levels and to check for potential side effects, as well as physical examinations.
The study involves several visits over a long period to monitor your health and how well the treatment is working. The exact number and frequency of visits will be explained by the study team. You will be closely monitored for quite some time after the treatment. You will also be asked to use reliable contraception throughout the study. The total duration of your participation in the study, including all follow-up, can be lengthy, potentially several years, to understand the long-term effects of the gene therapy.
Potential risks and benefits
Locations (13)
- Arkansas Children's Hospital - Hematology / OncologyVerified postcodeLittle Rock, United States
- C.S. Mott Children's Hospital - Hematology / OncologyVerified postcodeAnn Arbor, United States
- UW Health Carbone Cancer CenterVerified postcodeMadison, United States
- SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EADVerified postcodeSofia, Bulgaria
- CHU Rennes - Hopital PontchaillouVerified postcodeRennes, France
- APHP-Hopital Necker Enfants maladesVerified postcodeParis, France
- Universitätsklinikum des SaarlandesVerified postcodeHomburg, Germany
- Vivantes Klinikum im FriedrichshainVerified postcodeBerlin, Germany
- Academisch Medisch Centrum (AMC)Verified postcodeAmsterdam, Netherlands
- Erasmus Medisch CentrumVerified postcodeRotterdam, Netherlands
- Universitair Medisch Centrum GroningenVerified postcodeGroningen, Netherlands
- University Medical Center UtrechtVerified postcodeUtrecht, Netherlands
Common questions
What is haemophilia A?
Haemophilia A is a condition where your blood doesn't clot properly because you don't have enough of a protein called Factor VIII, which is essential for stopping bleeding.
What is gene therapy?
Gene therapy is a new type of treatment that aims to fix the root cause of a disease by introducing a healthy gene into your body to replace a faulty or missing one.
How is the treatment given?
The treatment, BAY 2599023 (DTX201), is given as a single infusion into a vein, similar to getting a drip.
Will I still need other Factor VIII treatments?
The goal of this therapy is to help your body produce its own Factor VIII, which could potentially reduce or eliminate the need for other treatments. However, this is what the study is aiming to find out.
How long will I be monitored?
Because this is a new type of treatment, participants will be monitored closely for an extended period, possibly several years, to understand the long-term safety and effectiveness.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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