Phase 3
N=28
Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01174446 ↗Enrolled (actual)
28
Serious AEs
5.5%
Results posted
Nov 2013
Primary outcome: Primary: Study Part 1- Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Per Dose — 14.30; 13.42 (IU·hr/dL) / (IU/kg)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- BAX 326 (Biological); BeneFIX (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- May 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Study Part 1- Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Per Dose |
14.30; 13.42 | — |
| SECONDARY Study Parts 1 and 3: Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity Per Dose (AUC0-∞/ Dose) |
16.07; 15.26; 17.38 | — |
| SECONDARY Study Parts 1 and 3: Mean Residence Time (MRT) |
28.93; 30.59; 29.04 | — |
| SECONDARY Study Parts 1 and 3: Clearance (CL) |
0.0622; 0.0655; 0.0576 | — |
| SECONDARY Study Parts 1 and 3: Incremental Recovery at Cmax (IR at Cmax) |
0.88; 0.73; 0.93 | — |
| SECONDARY Incremental Recovery (IR) at 30 Minutes Over Time |
0.78; 0.79; 0.83; 0.88; 0.89 | — |
| SECONDARY Change in Incremental Recovery (IR) at 30 Minutes Over Time |
0.03; 0.075; 0.06; 0.12 | — |
| SECONDARY Study Parts 1 and 3: Half Life (T 1/2) |
24.58; 26.28; 24.59 | — |
| SECONDARY Study Parts 1 and 3: Volume of Distribution at Steady State (Vss) |
1.72; 1.98; 1.74 | — |
| SECONDARY Study Part 2: Annualized Bleed Rate (ABR) During Treatment With BAX326 |
0.00; 0.00; 0.00; 0.00; 0.00; 1.99 | — |
| SECONDARY Bleeding Episodes Treated With 1, 2 or ≥3 Infusions of BAX326 by Bleeding Site and Cause |
65; 57; 122; 31; 84; 50 | — |
| SECONDARY Hemostatic Efficacy at Resolution of All Bleeding Episodes (BEs) Treated With BAX326 by Bleeding Site and Cause |
53; 32; 85; 17; 51; 40 | — |
| SECONDARY Total Weight-adjusted Dose Per Bleeding Episode (BEs) of All BEs Treated With BAX326 by Bleeding Site and Cause |
56.5; 59.0; 56.5; 68.7; 52.3; 70.0 | — |
| SECONDARY Consumption of BAX326 Per Event Per Participant |
50.5; 87.1 | — |
| SECONDARY Consumption of BAX326 Per Participant: Median Number of Infusions Per Month |
6.7; 2.7 | — |
| SECONDARY Consumption of BAX326 Per Participant: Median Weight-adjusted Consumption Per Month |
347.8; 167.3 | — |
| SECONDARY Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) |
— | — |
| SECONDARY Occurrence of Total Binding Antibodies of Indeterminate Specificity (Within Assay Variability) |
6; 0; 9 | — |
| SECONDARY Occurrence of Treatment Related Total Binding Antibodies |
0; 0; 0 | — |
| SECONDARY Number of Participants Who Experienced Severe Allergic Reactions (e.g. Anaphylaxis) |
— | — |
| SECONDARY Number of Participants Who Experienced Thrombotic Events |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Laboratory Parameters: Clinical Chemistry |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Laboratory Parameters: Hematology |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Laboratory Parameters: Vital Signs |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Laboratory Parameters: Thrombogenic Markers |
— | — |
| SECONDARY Number of Adverse Events (AEs) After BAX326 Treatment |
1; 0; 1; 0; 3; 0 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) After BAX326 Treatment |
1; 0; 1; 0; 2; 0 | — |
| SECONDARY EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Total Index Scores |
0.75; 0.75; 0.01; 0.72; 0.73; 0.00 | 0.7790 |
| SECONDARY EuroQoL (Quality of Life)-5 Dimensions Visual Analogue Scale (EQ-5D VAS) Scores |
58.75; 68.22; 9.98; 56.64; 62.07; 5.43 | 0.0056 sig |
| SECONDARY General Pain Assessment Through a Visual Analog Scale (VAS) |
32.67; 33.09; 0.35; 47.57; 39.93; -7.64 | 0.9059 |
| SECONDARY Short Form (36) Health Survey (SF-36): HRQoL 'Physical Component Score' (PCS) |
39.08; 41.35; 2.60; 37.38; 38.92; 1.54 | 0.0189 sig |
| SECONDARY SF-36: HRQoL 'Mental Health' (MH) |
47.53; 49.67; 2.01; 47.24; 45.03; -2.21 | 0.2009 |
| SECONDARY SF-36: HRQoL Physical Functioning' (PF) |
40.20; 40.75; 0.68; 40.04; 39.87; -0.16 | 0.5143 |
| SECONDARY SF-36: HRQoL Role-Physical (RP) |
40.39; 43.82; 3.47; 39.15; 37.45; -1.70 | 0.0162 sig |
| SECONDARY SF-36: HRQoL Role-Emotional |
44.22; 44.80; 0.37; 40.93; 39.43; -1.50 | 0.8210 |
| SECONDARY SF-36: HRQoL Bodily Pain |
42.09; 45.72; 3.45; 36.89; 39.33; 2.44 | 0.0146 sig |
| SECONDARY SF-36: HRQoL Mental Health |
45.52; 47.95; 2.44; 45.46; 42.64; -2.82 | 0.1258 |
| SECONDARY SF-36: HRQoL Vitality |
50.07; 52.75; 2.46; 50.17; 50.89; 0.72 | 0.1048 |
| SECONDARY SF-36: HRQoL Social Functioning |
41.80; 44.60; 2.78; 43.42; 42.17; -1.26 | 0.0663 |
| SECONDARY SF-36: HRQoL General Health |
37.84; 39.98; 2.20; 37.09; 39.07; 1.98 | 0.0562 |
| SECONDARY Pediatric Quality of Life Questionnaire (PedsQL) Physical Health Summary Score (Ages 12-16) |
65.63; 54.69; -10.94; 65.63; 65.63; 0.00 | 0.5792 |
| SECONDARY Pediatric Quality of Life Questionnaire (PedsQL) Psychosocial Health Summary Score (Ages 12-16) |
63.33; 55.83; -7.50; 88.33; 86.67; -1.67 | 0.4208 |
| SECONDARY Pediatric Quality of Life Questionnaire (PedsQL) Total Score (Ages 12-16) |
64.13; 55.43; -8.70; 80.43; 79.35; -1.09 | 0.5000 |
| SECONDARY Health-Related Quality of Life (HRQoL) Disease-specific: Haem-A-QoL |
40.68; 37.85; -3.52; 41.65; 41.37; -0.28 | 0.0633 |
| SECONDARY Health-Related Quality of Life (HRQoL) Disease-specific: Haemo-QoL - Participants On-Demand (Ages 12-16) |
40.00; 40.00; 0.00 | — |
| SECONDARY Health Resource Use - Number of Hospitalizations |
0.0; NA; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Health Resource Use - Total Days of Hospital Stay |
46.0; 23.0; 2.0; 5.5 | — |
| SECONDARY Health Resource Use - Emergency Room Visits |
0.0; NA; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Health Resource Use - Unscheduled Doctor's Office Visits |
0.0; NA; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Health Resource Use - Days Lost From Work or School |
0.0; NA; 0.0; 0.0; 0.0; 0.0 | — |
Summary
The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and moderately severe hemophilia B.
Eligibility Criteria
Main Inclusion Criteria:
- Participant is 12 to 65 years old at the time of screening
- Participant and/or legal representative has/have provided signed informed consent
- Participant has severe (factor IX (FIX) level 120 kg
- The participant has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s)
- The participant has a known hypersensitivity to hamster proteins or recombinant furin (rFurin)
- The participant has ongoing or recent evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
Data sourced from ClinicalTrials.gov (NCT01174446). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.