BAX 326 (rFIX) Continuation Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01286779
• Date of registration: 26/01/2011
• Prospective Registration: Yes
• Primary sponsor: Baxalta now part of Shire
• Public title: BAX 326 (rFIX) Continuation Study
• Scientific title: BAX 326 (Recombinant Factor IX): Evaluation of Safety, Immunogenicity, and Hemostatic Efficacy in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B - A Continuation Study
• Date of first enrolment: April 12, 2011
• Target sample size: 117
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01286779
• Study type: Interventional
• Study design: Allocation: Non-Randomized. 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, Germany, India, Ireland, Italy, Japan, Poland, Romania, Russian Federation, Spain, Sweden, Taiwan, Ukraine, United Kingdom

Contacts

• Name: Lydia Abad Franch, MD
• Affiliation: Shire

Key inclusion & exclusion criteria

Main Inclusion Criteria:

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

- Subject has completed Baxter clinical study 250901 (pivotal study) or Baxter clinical
study 251101 (pediatric study)

- Subject was 12 to 65 years old at the time of screening for Study 250901 or < 12 years
old at the time of screening for Study 251101

- Subject 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

- Subject has not developed an inhibitory FIX antibody during Baxter Pivotal Study
250901 or Pediatric Study 251101

Main Exclusion Criteria:

- Subject received factor IX product(s) other than BAX 326 upon completion of Baxter
Pivotal Study 250901 or Pediatric Study 251101

- Subject has been diagnosed with an acquired hemostatic defect other than hemophilia B

- For subjects transferring from Pivotal Study 250901: Subject's weight is < 35 kg or >
120 kg

- Subject is planned to take part in any other clinical study, with the exception of BAX
326 Surgery study as described in this protocol, during the course of the Continuation
Study



Age minimum: N/A
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hemophilia B

Intervention(s)

Biological: BAX 326 (Recombinant factor IX)

Primary Outcome(s)

Adverse Events Possibly or Probably Related to the Investigational Product [Time Frame: Assessed (based on patient diary) every 3 months until study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Secondary Outcome(s)

Consumption of BAX326: Weight Adjusted Consumption Per Bleeding Episode [Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Annualized Bleed Rate During Prophylaxis Treatment [Time Frame: For prophylactic treatment the period from first to last prophylactic infusion is considered.]

Development of Inhibitory and Total Binding Antibodies to Factor IX [Time Frame: Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.]

Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire SF-36 [Time Frame: Baseline at exposure day 1 and at study completion/termination.]

Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution [Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Pharmacokinetics: Mean Residence Time (MRT) [Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours]

Pharmacokinetics: Volume of Distribution at Steady State (Vss) [Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours]

Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed [Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Pharmacokinetics: Incremental Recovery (IR) Over Time [Time Frame: IR over time was measured as Baseline and at Completion/Termination visit within 30 minutes pre-infusion and at 30 (± 5) minutes post-infusion.]

Development of Antibodies to Chinese Hamster Ovary Proteins (CHO Proteins) and rFurin [Time Frame: Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.]

Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire EQ-5D and Pain Score. [Time Frame: Baseline at exposure day 1 and at study completion/termination.]

Pharmacokinetics: Elimination Phase Half-life (T1/2) [Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours]

Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire Haemo-QoL and Haem-A-QoL [Time Frame: Baseline at exposure day 1 and at study completion/termination.]

Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year [Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Occurrence of Severe Allergic Reactions and Thrombotic Events [Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-8) [Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours]

Clinical Significant Changes in Routine Laboratory Parameters and Vital Signs [Time Frame: Measurements at screening and at study completion/termination are included in the analysis.]

Consumption of BAX 326: Number of Infusions Per Month and Per Year [Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).]

Changes in Health Related Quality of Life Using the Peds QL [Time Frame: Baseline at exposure day 1 and at study completion/termination.]

Pharmacokinetics: Incremental Recovery (IR) [Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours.]

Pharmacokinetics: Systemic Clearance (CL) [Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours]

Secondary ID(s)

2010-022726-33

251001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/09/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01286779