A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A

  • STATUS
    Recruiting
  • End date
    Aug 12, 2024
  • participants needed
    48
  • sponsor
    Bayer
Updated on 8 January 2022

Summary

The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A. In addition, pharmacokinetic parameters of Kovaltry will be assessed in a subset of patients.

Details
Condition Hemophilia A
Treatment Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3
Clinical Study IdentifierNCT04565236
SponsorBayer
Last Modified on8 January 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Part A (PTPs)
Chinese participants with severe hemophilia A (defined as Factor VIII (FVIII): C < 1% with one- stage clotting assay documented at the time of screening)
Currently receiving on-demand or any type of prophylaxis treatment regimen with any FVIII product
For participants < 12 years of age, ≥ 50 exposure days (ED); for participants ≥ 12 to 65 years of age, ≥ 150 ED with any FVIII product
No current evidence of inhibitor
No history of FVIII inhibitor formation
Signed informed consent
Part B (PUPs/MTPs)
Participants must be <6 years of age at the time of their parent or legal representative's signature of informed consent on the participant's behalf
Chinese participants with severe hemophilia A (defined as Factor VIII (FVIII): C < 1% with one- stage clotting assay documented at the time of screening)
PUPs must have no previous exposure to any FVIII product. MTPs must have no more than 1 ED with any purified FVIII concentrate or 3 exposures with FFP or cryoprecipitate
MTPs must have no current evidence of inhibitor antibody as measured by the Nijmegen-modified Bethesda assay (<0.6 BU/mL) in 2 consecutive samples and must have absence of clinical signs or symptoms of decreased response to FVIII administration. Testing for the 2 negative samples must be performed by the central laboratory at least 1 week but not more than 2 weeks apart. Participants may not receive FVIII product within 72 hours prior to the collection of samples for inhibitor testing
PUPs and MTPs must observe a 6-month washout period if they have received subcutaneous factor substitution therapy (emicizumab)
PUPs may be included if they will receive their first FVIII dose with KOVALTRY for treatment of first bleed and agree to start prophylaxis as part of their care. MTPs may be included if they agree to start prophylaxis as part of their care

Exclusion Criteria

Part A (PTPs)
Any other bleeding disease that is different from hemophilia A (e.g. von Willebrand disease, hemophilia B)
Platelet count < 100 000/mm^3
Impaired renal function (serum creatinine > 2.0 mg/dL) or active liver disease (alanine aminotransferase/aspartate aminotransferase [ALT/AST] > 5x ULN)
Human immunodeficiency virus (HIV) positive with an absolute CD4 lymphocyte cell count < 250 cells/μL
Known hypersensitivity to the active substance, mouse or hamster protein
Receiving 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
Requiring any pre-medication to tolerate FVIII infusions (e.g. antihistamines)
Currently participating in another investigational drug study, or having previously participated in a clinical study involving an investigational drug within 30 days of signing informed consent or participated in completed interventional clinical studies with BAY81-8973 (Kovaltry)
Planned major surgery, defined as surgery with respiratory assistance and/or general anesthesia
Part B (PUPs/MTPs)
Any other bleeding disease that is different from hemophilia A (e.g. von Willebrand disease, hemophilia B)
Platelet count < 100 000/mm^3
Impaired renal function (serum creatinine >2× upper limit of normal [ULN]) or active liver disease (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >5× ULN) based on screening laboratory assessments
MTPs with history of FVIII inhibitor formation
Known hypersensitivity to the active substance, mouse or hamster protein
First treatment with KOVALTRY for high risk bleeding situations (e.g., surgery, intracranial bleed) or requiring intensive or prolonged treatment
Receiving 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
Requiring any pre-medication to tolerate FVIII infusions (e.g. antihistamines)
Currently participating in another investigational drug study, or having previously participated in a clinical study involving an investigational drug within 30 days of signing informed consent or participated in completed interventional clinical studies with BAY 81-8973 (Kovaltry)
Planned major surgery, defined as surgery with respiratory assistance and/or general anesthesia
Unable to tolerate volume of blood draws required for study participation
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