Prevention of excessive bleeding in haemophilia A or B subjects with inhibitors undergoing major surgical procedures
Conditions
Interventions
Trade Name: CEVENFACTA
Pharmaceutical Form:
INN or Proposed INN: Eptacog beta (activated)
Current Sponsor code: LR769
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Conce
Sponsors
LFB Biotechnologies
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - be male with a diagnosis of congenital hemophilia A or B of any severity - have one of the following: a. current positive inhibitor test BU =5 (as confirmed at screening by the institutional lab) or history of high-responding inhibitors (BU =5) not further successfully treated by Immune Tolerance Induction OR b. a condition precluding the use of FVIII or FIX products to treat or prevent bleeding such as a previous anamnestic response after exposure to factor concentrates or a previous failure to respond to FVIII or FIX concentrates - be =12 years to =65 years of age - be scheduled for an elective major surgical procedure - have Hb =12 g/dL - patient's and/or parent(s)'/legal guardian(s)' understanding and willingness to comply with the conditions of the protocol written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 2 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - have any coagulation disorder other than hemophilia A or B be immunosuppressed (CD4+ cell counts at screening should be 200/µL) - known intolerance to Eptacog Beta (activated) or any of its excipients - currently receiving immune tolerance induction (ITI) therapy - have a known or suspected allergy or hypersensitivity to rabbits or rabbit proteins - have platelet count 3 times the upper limit of normal [ULN]) and/or renal impairment (creatinine >2 times the ULN) - have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, DVT, or PE) within 2 years prior to the planned first dose of Eptacog Beta (activated), uncontrolled arrhythmia, or current NYHA functional classification score of stages II – IV - have an active malignancy (those with non-melanoma skin cancer are allowed) - have any life-threatening disease or other disease or condition which, according to the investigator’s judgment, could imply a potential hazard to the patient, or interfere with the study participation or study outcome (e.g. chronic, unmanaged hepatitis infection) - be using aspirin, non-steroidal anti-inflammatory drugs (NSAIDS), herbs, natural medications, or other drugs with platelet inhibitory properties within one week prior to surgery and for the duration of treatment with Eptacog Beta (activated) - have active gastric or duodenal ulcer disease - have received a FVII- or FVIIa-containing product (either plasma derived or recombinant) within 24 hours prior to administration of Eptacog Beta (activated) - have a contraindication to antifibrinolytics - have planned combined major surgeries at the same time or have already been enrolled and treated for a previous elective major surgery in the same SCOPE HIM study - be administered pharmacologic thromboprophylaxis within 5 half-lives of that medication before surgery or for the duration of treatment with Eptacog Beta (activated)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assess the efficacy of Eptacog Beta (activated) in terms of achieving and maintaining hemostasis in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX undergoing elective major surgical procedures;Secondary Objective: Assess the safety of Eptacog Beta (activated);Primary end point(s): Proportion of successfully treated major procedures defined by a good or excellent global hemostatic response obtained by summing assigned scores from a defined 4-point hemostatic scale determined at wound closure (T0), at 24 hours after surgical wound closure, and at 120 hours after surgical wound closure.;Timepoint(s) of evaluation of this end point: At wound closure (T0) (based on data collected during the intraoperative period) At 24 hours after surgical wound closure (based on data collected during the 24-hour postoperative period, i.e. 0-24 hours period) At 120 hours after surgical wound closure (based on data collected during the 120-hour postoperative period, i.e. 0-120 hours) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Proportion of successfully treated major procedures defined by a good or excellent hemostatic response based on the 4-point hemostatic scale at each of the reference timepoints - Proportion of successfully treated major procedures defined by a good or excellent global hemostatic response based on the 4-point hemostatic scale as assessed by the investigator at subsequent days - Mean difference between actual measurable intraoperative blood loss and maximum predicted blood loss in a similar patient without a bleeding disorder for the same major procedure - Mean difference between actual measurable pstoperative blood loss (measured by the amount of drainage in mL where, and for the duration, drain is needed) and maximum predicted blood loss in a similar patient without a bleeding disorder for the same major procedure at each of the reference timepoints - Mean difference between actual blood product transfusion and maximum predicted blood product transfusion in mL in a similar patient without a bleeding disorder for the same major procedure at each of the reference timepoints - Mean change in Hemoglobin level from initiation of the surgery until last visit - Mean amount of Eptacog Beta (activated) used to maintain hemostasis during surgery and post-surgery - Mean number of bleeding episodes at the surgical site between surgical wound closure and last visit - Mean number of surgical interventions/re-explorations for bleeding episodes between surgical wound closure and last visit - Number of deaths from bleeding attributed to ineffective hemostasis - Number and incidence of TEAEs (i.e. all AEs that occur at the time of or after the IMP administration until follow-up phone call) and AESIs (i.e. thromboembolic events and hypersensitivity reactions) - Mean value at each timepoint and mean change from baseline to each post-baseline timepoint in clinical laboratory parameters (continuous); proportion for each response category at each timepoint and pr | — |
Countries
Malaysia, Mexico, South Africa, Thailand, Türkiye, United States
Contacts
Public ContactGlobal Clinical Development Leader
LFB Biotechnologies
Outcome results
None listed