Congenital haemophilia with inhibitors
Conditions
Sponsors
KM Biologics Co., Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1)Congenital haemophilia A or B patients with inhibitors to factor VIII or factor IX. (2)Patients who or whose legally acceptable representatives have provided written informed consent.
Exclusion criteria
Exclusion criteria: (1) Pediatric patients (aged 18 years or younger) for Part 1 (2) Patients undergoing immune tolerance induction (ITI) treatment (3) Patients with diseases that develop abnormality in the coagulation/fibrinolytic system other than the target disease of the study or suspected of such (4) Patients with hypercoagulability or such possibilities (5) Patients with a history of disseminated intravascular coagulation (DIC) or thromboembolism (6) Patients with a history of shock or hypersensitivity to protein preparations such as blood derivatives (7) Patients who have undergone surgical procedure and have not fully recovered when participating in the study or patients who are scheduled to undergo surgery during the study (8) Patients with acquired immunodeficiency syndrome (AIDS) defining illness (9) Patients with severe liver disorder or renal disorder (10) Patients with current serious medical conditions such as malignant tumor or leukaemia who will or are highly likely to undergo chemotherapy or radiotherapy during the study (11) Patients with hemolytic or blood loss anaemia (12) Patients who have participated in any other clinical study and received any investigational product within 1 month prior to the informed consent for the study or patients who are participating, or who are scheduled to participate during the study, in any other clinical study at the time of informed consent for the study (13) Patients who have received HEMLIBRA (R) Subcutaneous Injection within 4 months prior to the informed consent for the study (14) Patients who are considered ineligible for the study by the investigator or subinvestigator for any other reason
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Annual bleeding rate safety Number of cases, number of patients, and incidence rate of adverse events | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy 1) Annual bleeding rate by groups divided by demographics 2) Annual bleeding rate by bleeding site 3) Time for treatment 4) HRQoL and health conditions other 1) PK/PD analysis from the initial to 4th doses in the regular treatment period 2) PK/PD assessment every 4 weeks in the regular treatment period | — |
Countries
Japan
Contacts
Public ContactYasuhiko Shinmura
KM Biologics Co., Ltd.
Outcome results
None listed