Lead-in study to collect prospective efficacy and quality-of-life data of current treatment in haemA
Research type
Research Study
Full title
A prospective, lead-in study to collect bleeding episodes, Factor VIII (FVIII) infusions, and patient-reported outcomes in patients with hemophilia A
IRAS ID
1003676
Contact name
Sylvia Hiege
Contact email
Sponsor organisation
Bayer AG
Eudract number
2019-004480-48
Research summary
Haemophilia A is an inborn condition where the body does not create enough of a protein called clotting factor 8 (FVIII) in the blood. People with haemophilia A may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. In severe haemophilia A (FVIII levels equal or less than 1%) bleeding episodes are more likely.
In this “lead-in study” patients will receive their standard treatment and no study treatment will be given. Researchers will collect data about the patients’ bleeding episodes, their prescribed medications containing FVIII, additional FVIII use to treat bleeding episodes and how they feel about their symptoms and pain.
After completion of this study, participants can join the gene therapy study with the new treatment BAY2599023 if they fulfill the criteria for participation. BAY2599023 delivers the human FVIII gene into the body by using 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. Researchers will compare the information collected from the 2 studies (lead-in study vs gene therapy study). This will allow them to compare the new treatment to the existing standard treatment.
Patients with severe haemophilia A who have previously received treatments containing FVIII for at least 150 days in their lives and who have used FVIII to prevent bleeding (prophylaxis) for at least 6 consecutive months before joining the study will be able to participate.
There will be 5 visits in which doctors will:
• take blood samples
• perform a liver ultrasound scan
• check the patients’ health
• ask about the treatment they are using
There will also be monthly phone calls.
The patients will be in this study for at least 6 months before they can start the gene therapy study. If they decide not to start, they will have 1 more study visit and will continue to have monthly phone calls for a maximum of an additional 6 months.REC name
Wales REC 5
REC reference
21/WA/0274
Date of REC Opinion
20 Sep 2021
REC opinion
Favourable Opinion