Phase 3
Completed N=22
Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures
Source: ClinicalTrials.gov NCT01913405 ↗Enrolled (actual)
22
Serious AEs
9.1%
Results posted
May 2018
Primary outcomePrimary: Global Hemostatic Efficacy Assessment Score (GHEA) - Composed of 3 Individual Ratings — 100.0; 100.0; 100.0; 100.0 Percentage of surgeries
◆ Published Evidence
Established
29citations · ~3 / year
Efficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patients.
Summary
The purpose of the study is to evaluate the efficacy and safety of BAX 855 in severe hemophilia A previously treated (PTP) males, 12 to 65 years of age who are undergoing elective surgical or other invasive procedures.
Linked Publications
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Efficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patients.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Global Hemostatic Efficacy Assessment Score (GHEA) - Composed of 3 Individual Ratings |
100.0; 100.0; 100.0; 100.0; 100.0; 100.0 | — |
| SECONDARY Intraoperative Blood Loss |
10; 10; 4.5; 5; 20; 150 | — |
| SECONDARY Postoperative Blood Loss |
10; 750; 1; 0; 27.5; 213.5 | — |
| SECONDARY Overall Perioperative Blood Loss |
50; 246; 5.5; 9; 40; 675 | — |
| SECONDARY Transfusion Requirements |
438.0; 384.0; 600.0 | — |
| SECONDARY Occurrence of Bleeding Episodes and Additional Need for Surgical Intervention |
2; 0; 1; 1; 1; 1 | — |
| SECONDARY Consumption of BAX855 |
562.076; 746.103; 507.881; 122.673; 62.492; 69.442 | — |
| SECONDARY Pharmacokinetics (PK) - Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-∞) |
2743.3; 3201.8 | — |
| SECONDARY Pharmacokinetics (PK) - Area Under the Plasma Concentration/Time Curve From Time 0 to 96 Hours Post-infusion (AUC0-96h) |
2701.3; 3153.7 | — |
| SECONDARY Pharmacokinetics (PK) - Terminal Half-life (T1/2) |
14.63; 14.53 | — |
| SECONDARY Pharmacokinetics (PK) - Mean Residence Time (MRT) |
19.26; 18.68 | — |
| SECONDARY Pharmacokinetics (PK) - Clearance (CL) |
0.02347; 0.02042 | — |
| SECONDARY Pharmacokinetics (PK) - Apparent Volume of Distribution at Steady State (Vss) |
0.4316; 0.3673 | — |
| SECONDARY Pharmacokinetics (PK) - Incremental Recovery(IR) |
2.106; 2.721; 2.123; 2.677 | — |
| SECONDARY Development of Inhibitory Antibodies to Factor VIII (FVIII) |
— | — |
| SECONDARY Development of Treatment Emerging Binding Antibodies to Factor VIII (FVIII), Treatment Emergent Binding Antibodies to PEGylated Recombinant FVIII (BX855), and Treatment Emerging Binding Antibodies to Polyethylene Glycol (PEG) |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Development of Treatment Emerging Anti-chinese Hamster Ovary (CHO) Antibodies |
— | — |
| SECONDARY Occurrence of Thrombotic Events |
— | — |
| SECONDARY Incidence of Severe Allergic Reactions (e.g. Anaphylaxis) |
— | — |
| SECONDARY Other Investigational Product (IP) - Related Adverse Events |
2 | — |
| SECONDARY Clinically Significant Changes in Vital Signs - Body Temperature |
36.60; 36.60; 0.00 | — |
| SECONDARY Clinically Significant Changes in Vital Signs - Systolic and Diastolic Blood Pressure (BP) |
120; 115.0; -5.0; 75.0; 75.0; 0.0 | — |
| SECONDARY Clinically Significant Changes in Vital Signs - Respiratory Rate |
14.0; 14.0; 0.0 | — |
| SECONDARY Clinically Significant Changes in Vital Signs - Pulse Rate |
70.0; 70.0; -1.0 | — |
| SECONDARY Clinically Significant Changes in Routine Laboratory Parameters- Hematology and Chemistry |
1; 1; 1; 1; 1 | — |
Eligibility Criteria
Inclusion Criteria
- Participant requires an elective major or minor surgical, dental or other invasive procedure (e.g. biopsy, endoscopy).
- Participant and/or legal representative has/have provided signed informed consent.
- Participant has severe hemophilia A (Factor VIII (FVIII) level 2.0 mg/dL), as confirmed by central laboratory at screening.
- Participant has severe chronic hepatic dysfunction (eg ≥5 X upper limit of normal alanine aminotransferase (ALT), as confirmed by the central laboratory at screening, or a documented International Normalized Ratio (INR) > 1.5).
- Participant has a known hypersensitivity towards mouse or hamster proteins, polysorbate 80 or to PEG.
- Participant is currently using or has recently ( 10 mg/day, or alpha interferon) other than anti-retroviral chemotherapy.
- Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
Data sourced from ClinicalTrials.gov (NCT01913405) and the linked publication. 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. Informational only — not medical advice.