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

Study Identifier:
19855
ClinicalTrials.gov Identifier:
NCT04565236
EudraCT Identifier:
2021-003537-11
Recruiting

Study Details

Medical Condition
  • Hemophilia A
Study Drug
  • Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1
  • Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2
  • Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3
Date
Sep 2020 - Sep 2028
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 0 - 65 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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

Protocol Summary

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Trial Locations

Location
Status
Location
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, China, 510515
Status
Withdrawn
Location
Shenzhen Children's Hospital
Shenzhen, Guangdong, China, 518017
Status
Not yet recruiting
Location
Shijiazhuang General Hospital
Shijiazhuang, Hebei, China, 050000
Status
Completed
Location
NJ Drum Tower Hospital, the Affil Hos of NJ Univ Med School
Nanjing, Jiangsu, China, 210008
Status
Completed
Location
1st Affiliated hospital of Soochow University
Suzhou, Jiangsu, China, 215000
Status
Completed
Location
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, China, 330006
Status
Completed
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