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A Phase 3 safety study of BAY 94-9027 in children 7 to <12 years of age with severe hemophilia A
EU CTIS ID: 2023-504388-18-00
What this study is testing
To assess safety and tolerability of BAY 94-9027 replacement therapy in previously treated patients 7 to <12 years of age with severe hemophilia A
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participant must be 7 to <12 years of age at the time of parent / guardian signing the informed consent.
- Participants with known medical history of severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%. FVIII:C) based on reliable prior documentation in clinical records of the participants. If no reliable documentation is available, FVIII activity must be measured at the time of screening after a 48-72 hours wash-out period (depending on his previous product).
- Male
- Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent.
- Participant has understood the study and if appropriate for his age, has signed an informed assent. The parent(s) or guardian(s) is capable of giving signed informed consent and are able to comply with the requirements and restrictions listed in the informed consent form (ICF) and the protocol.
- Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study.
You likely can't join if
- "History of FVIII inhibitors. Inhibitor to FVIII is defined as a titer >0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. Participants with a maximum historical titer of <1.0 BU on no more than 1 occasion with the classical Bethesda assay but at least 3 subsequent negative results [<0.6 BU] are eligible."
- Planned major surgery during the study
- Any individual who is currently receiving or received chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months
- Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within the last 6 months
- The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug within 30 days of study entry or previous participation in a clinical study with BAY 94-9027.
- Current evidence of inhibitor to FVIII measured using the Nijmegen modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Participants should not receive FVIII within 48 h prior to the collection of screening samples and should have FVIII administered within the prior 2-3 weeks
See the full eligibility criteria
Who can join
- Participant must be 7 to <12 years of age at the time of parent / guardian signing the informed consent.
- Participants with known medical history of severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%. FVIII:C) based on reliable prior documentation in clinical records of the participants. If no reliable documentation is available, FVIII activity must be measured at the time of screening after a 48-72 hours wash-out period (depending on his previous product).
- Male
- Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent.
- Participant has understood the study and if appropriate for his age, has signed an informed assent. The parent(s) or guardian(s) is capable of giving signed informed consent and are able to comply with the requirements and restrictions listed in the informed consent form (ICF) and the protocol.
- Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study.
What rules you out
- "History of FVIII inhibitors. Inhibitor to FVIII is defined as a titer >0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. Participants with a maximum historical titer of <1.0 BU on no more than 1 occasion with the classical Bethesda assay but at least 3 subsequent negative results [<0.6 BU] are eligible."
- Planned major surgery during the study
- Any individual who is currently receiving or received chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months
- Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within the last 6 months
- The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug within 30 days of study entry or previous participation in a clinical study with BAY 94-9027.
- Current evidence of inhibitor to FVIII measured using the Nijmegen modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Participants should not receive FVIII within 48 h prior to the collection of screening samples and should have FVIII administered within the prior 2-3 weeks
- Any other inherited or acquired bleeding disorder in addition to hemophilia A (e.g., von Willebrand disease, hemophilia B)
- Platelet count <100,000 cells/μL
- Serum creatinine > 2x upper limit of normal
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5x upper limit of normal
- Known hypersensitivity or allergic reaction to drug substance, excipients or mouse or hamster protein
- Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study
- Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Brazil
- Turkey
- Argentina
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Brazil; Turkey; Argentina; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.