Congenital Haemophilia A with Factor VIII Inhibitors MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850
Conditions
Interventions
Trade Name: OBIZUR
Product Name: OBIZUR
Product Code: BAX802
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Susoctocog alfa
Current Sponsor code: BAX802
Other
Sponsors
Baxalta Innovations GmbH
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Subject requires a major or minor elective surgical, dental or other invasive procedure 2. Subject is male and = 12 to = 75 years old at the time of screening 3. Subject has provided signed informed consent (and assent for adolescent subjects, as applicable) in accordance with local regulatory requirements 4. Subject has severe (FVIII level =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1. The subject requires emergency surgery 2. Severe chronic liver dysfunction or disease (e.g., = 5 x upper limit of normal [ULN] alanine aminotransferase [ALT], as confirmed by central laboratory at screening or a documented prothrombin time/international normalized ratio [PT/INR] > 1.5) 3. Clinically symptomatic renal disease (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening 4. Anti-pFVIII inhibitor > 10 BU prior to surgery 5. Platelet count < 100,000/µL at screening 6. Subject has another active coagulation disorder other than haemophilia A, as per the medical history 7. Planned use of a-interferon with or without ribavarin for HCV infected patients or planned use of a protease inhibitor for HIV infected patients. Patients currently taking any of these medications for = 30 days are eligible 8. Known hypersensitivity to rpFVIII, or hamster or murine proteins 9. Subject has an ongoing or recent (within 3 months of screening) thrombo-embolic disease, fibrinolysis or disseminated intravascular coagulation (DIC) 10. Subject has been exposed to an IP within 30 days prior to enrolment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study 11. Subject is unable to tolerate quantity of blood to be drawn for protocol procedures 12. Subject is a family member or employee of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to evaluate the perioperative hemostatic efficacy of BAX 802 in male subjects with CHA with inhibitors to hFVIII undergoing major or minor elective surgical, dental, or other invasive procedures as determined by the Global Hemostatic Efficacy Assessment (GHEA) score.;Secondary Objective: 1. To determine the safety of BAX 802 in the perioperative setting by assessing: -The development of and change in titer of anti-porcine FVIII (pFVIII) and anti-hFVIII antibodies (binding and neutralizing), and development of binding antibodies to baby hamster kidney (BHK) proteins -The occurrence of thrombo-embolic events and/or allergic reactions to BAX 802 -The occurrence of adverse events (AEs) related to BAX 802 -The occurrence of clinically significant changes in vital signs and routine laboratory parameters related to BAX 802 2. To determine the intraoperative, postoperative, and overall perioperative blood loss, compared to the estimated volume of expected average and maximum blood loss in a comparable healthy individual as predicted preoperatively by the Investigator/surgeon 3. To determine the proportion of major surgeries with good or excellent hemostatic score 4. To determine the daily and total weight-adjusted administration of BAX 802 per subject;Primary end point(s): The primary outcome measure is the proportion of all surgical, dental, or other invasive procedures with a "good" or "excellent" response as measured by GHEA score which is composed of 3 individual ratings: -GHEA1: Assessment of intraoperative (Day 0) hemostatic efficacy of BAX 802 performed by the operating surgeon at the end of surgery. -GHEA2: Assessment of postoperative hemostatic efficacy of BAX 802 at postoperative Day 1 (approximately 24 hours [+/- 6 hours] postsurgery) performed by the operating surgeon. Note: If a patient is discharged <24 hours following surgery, then the GHEA2 hemostatic efficacy assessment will require a return visit the f | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Varying times as defined by the protocol.;Secondary end point(s): Efficacy 1. Intra- and post-operative blood loss compared to the estimated volume of expected average blood loss and expected maximum blood loss in a comparable healthy individual with similar demographic characteristics as predicted preoperatively by the investigator/surgeon at the following time points: • Intraoperative, from start until the end of surgery • Postoperative Day 1, from end of surgery to approximately 24 hours (+/- 6 hours) after surgery • Overall perioperative at discharge or 24 to 72 hours after the last perioperative treatment dose of BAX 802 (whichever is earlier) 2. Proportion of major surgeries with good or excellent hemostatic score 3. Daily and total weight-adjusted administration of BAX 802 per subject 4. Amount of blood products (e.g., Whole blood, red blood cells, platelets, and plasma transfused) Safety 1. Development of, and changes to, the titer of inhibitory and binding antibodies (IgG and IgM) to pFVIII 2. Development of, and changes to, the titer of inhibitory and binding antibodies (IgG and IgM) to hFVIII 3. Development of binding antibodies to BHK proteins 4. Occurrence of thrombo-embolic events 5. Incidence of severe allergic reactions (e.g., anaphylaxis) 6. Incidence of other IP-related AEs 7. Incidence of clinically significant changes in vital signs and routine laboratory parameters (hematology, clinical chemistry) | — |
Countries
Bulgaria, Canada, Germany, Italy, Netherlands, Norway, Poland, Russian Federation, South Africa, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Development
Baxalta Innovation GmbH
Outcome results
None listed