Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With… (NCT03588299) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
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"
United States, Bulgaria, France11 participantsStarted 2018-11-07
Plain-language summary
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.
Who can participate
Age range
18 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Males age 18 years or older.
* Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels \< 1% of normal or at screening.
* Have \>150 exposure days (EDs) to FVIII concentrates (recombinant or plasma-derived).
If on prophylaxis, are required to be willing to stop prophylactic treatment at specified time points throughout the study or If on-demand: should have had \> 4 bleeding events in the last 52 weeks
\- Agree to use reliable barrier contraception.
Exclusion Criteria:
* History of allergic reaction to any FVIII product.
* Clinically relevant findings in the physical examination considered critical by the treating physician, including obesity with BMI \> 35 kg/m\*2
* Current evidence of measurable inhibitor against factor VIII, prior history of inhibitors to FVIII protein or clinical history suggestive of inhibitor.
* Evidence of active hepatitis B or C.
* Currently on antiviral therapy for hepatitis B or C.
* Significant underlying liver disease.
* Serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm\*3; HIV+ and stable participants with CD4 count \>200/mm\*3 and undetectable viral load are eligible to enroll.
* Detectable antibodies reactive with AAVhu37capsid.
* Participant with another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B).
* Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an invest…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest