BAX 326 Surgery Study in Hemophilia B Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01507896
• Date of registration: 09/01/2012
• Prospective Registration: No
• Primary sponsor: Baxalta now part of Shire
• Public title: BAX 326 Surgery Study in Hemophilia B Patients
• Scientific title: BAX 326 (Recombinant Factor IX): A Phase 3 Prospective, Multicenter Study Evaluating Efficacy and Safety in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level 1-2%) Hemophilia B Undergoing Surgical or Other Invasive Procedures
• Date of first enrolment: December 19, 2011
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01507896
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Brazil, Bulgaria, Chile, Colombia, Czech Republic, Czechia, Japan, Poland, Romania, Russian Federation, Sweden, Ukraine, United Kingdom

Contacts

• Name: Study Director
• Affiliation: Shire

Key inclusion & exclusion criteria

Main Inclusion Criteria:

- Participant and/or legal representative has/have voluntarily provided signed informed
consent.

- Participant has severe (FIX level < 1%) or moderately severe (FIX level 1-2%)
hemophilia B (based on the one stage activated partial thromboplastin time (aPTT)
assay), as tested at screening at the central laboratory.

- Participant requires surgery

- Participant has previously been treated with plasma-derived and/or recombinant FIX
concentrate(s) for a minimum of 150 exposure days

- Participant has no evidence of a history of FIX inhibitors

- Participant is immunocompetent as evidenced by a CD4 count = 200 cells/mm3.

- Participant is human immunodeficiency (HIV) negative or is HIV+ with a viral load <
200 particles/µL ~ < 400,000 copies/mL.

Main Exclusion Criteria:

- Participant has a history of FIX inhibitors with a titer = 0.6 Bethesda Units (BU) (as
determined by the Nijmegen modification of the Bethesda assay or the assay employed in
the respective local laboratory) at any time prior to screening.

- Participant has a detectable FIX inhibitor at screening, with a titer =0.6 Bethesda
Units (BU) as determined by the Nijmegen modification of the Bethesda assay in the
central laboratory.

- Participant has a history of allergic reaction or evidence of an ongoing or recent
thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).

- Known hypersensitivity to hamster proteins or recombinant furin.

- Evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated
intravascular coagulation (DIC).

- Abnormal renal function

- Severe chronic liver disease

- Active hepatic disease with ALT or AST levels > 5 times the upper limit of normal.

- Diagnosis of an iherited or acquired hemostatic defect other than hemophilia B.

- Platelet count < 100,000/mL.



Age minimum: 12 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hemophilia B

Intervention(s)

Biological: Recombinant factor IX

Primary Outcome(s)

Daily Weight-Adjusted Dose of BAX326 Per Participant [Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery)]

Actual Postoperative Blood Loss Compared to Average and Maximum Blood Loss Predicated Preoperatively by the Operating Surgeon [Time Frame: At postoperative day 3 (approximately 72 hours postoperatively)]

Actual Intraoperative Blood Loss [Time Frame: On day of surgery]

Pre-Surgical Pharmacokinetics (PK): Mean Residence Time (MRT) [Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr]

Postoperative Hemostatic Efficacy on Day of Discharge [Time Frame: At discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery)]

Total Weight-Adjusted Dose of BAX326 Per Participant [Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery)]

Safety: Number of Adverse Events Related to BAX326 [Time Frame: Throughout the study period (approximately 2 years 5 months)]

Actual Intraoperative Blood Loss Compared to Average and Maximum Blood Loss Predicted Preoperatively by the Operating Surgeon [Time Frame: On day of surgery]

Number of Units of Blood Product Transfused [Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery)]

Pre-Surgical Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve Per Dose (Total AUC/Dose) [Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr]

Pre-Surgical Pharmacokinetics (PK): Incremental Recovery (IR) at 30 Min [Time Frame: Within 30 mins pre-infusion and post-infusion at 30 minutes]

Safety: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX) [Time Frame: Throughout the study period (approximately 2 years 5 months)]

Pre-Surgical Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) [Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr]

Safety: Occurence of a Thrombotic Event [Time Frame: Throughout the study period (approximately 2 years 5 months)]

Incremental Recovery (IR) at 15±5 Minutes Following Loading Dose Prior to Surgery [Time Frame: Within 60 minutes prior to surgery and 15 ± 5 minutes after loading dose/rebolus, if applicable.]

Postoperative Hemostatic Efficacy at Drain Removal [Time Frame: At drain removal (from 1-3 days postoperatively)]

Volume of Blood Product Transfused [Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery)]

Actual Postoperative Blood Loss [Time Frame: At drain removal (from 1-3 days postoperatively)]

Pre-Surgical Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL) [Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr]

Safety: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) [Time Frame: Throughout the study period (approximately 2 years 5 months)]

Intraoperative Hemostatic Efficacy [Time Frame: On day of surgery]

Postoperative Hemostatic Efficacy at Postoperative Day 3 [Time Frame: At postoperative day 3 (approximately 72 hours postoperatively)]

Pre-Surgical Pharmacokinetics (PK): Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to 72 Hours Post-infusion Per Dose [Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr]

Pre-Surgical Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2) [Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr]

Secondary ID(s)

2011-000413-39

251002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/02/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01507896