Phase 3
Completed N=66
BAX 855 Pediatric Study
Source: ClinicalTrials.gov NCT02210091 ↗Enrolled (actual)
66
Serious AEs
3.1%
Results posted
Dec 2016
Primary outcomePrimary: Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII) — 0; 0; 0 participants
◆ Published Evidence
Established
80citations · ~9 / year
Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A.
Summary
The study purpose is:
* To assess the incidence of FVIII inhibitory antibodies during 6 months of twice weekly prophylactic treatment with BAX 855 or 50 exposure days (EDs), whichever occurs last.
* To compare pharmacokinetic (PK) parameters to ADVATE.
* To assess hemostatic efficacy in prophylaxis and the treatment of bleeding episodes.
* To evaluate safety and immunogenicity.
Linked Publications
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Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII) |
0; 0; 0 | — |
| SECONDARY Annualized Bleeding Rate (ABR) |
2.37; 3.75; 3.04; 0.862; 1.355; 1.103 | — |
| SECONDARY Consumption of BAX 855: Number of Prophylactic Infusions Per Month Per Participant |
8.07; 7.72; 7.89 | — |
| SECONDARY Consumption of BAX 855: Number of Prophylactic Infusions Per Year (Annualized) Per Participant |
96.82; 92.61; 94.65 | — |
| SECONDARY Consumption of BAX 855: Weight-adjusted Dose of Prophylactic Infusions Per Month Per Participant |
458.93; 455.86; 457.35 | — |
| SECONDARY Consumption of BAX 855: Weight-adjusted Dose of Prophylactic Infusions Per Year (Annualized) Per Participant |
5507.20; 5470.32; 5488.20 | — |
| SECONDARY Consumption of BAX 855: Number of Infusions Per Bleeding Episode |
1.17; 1.40; 1.30 | — |
| SECONDARY Consumption of BAX 855: Weight-adjusted Dose Per Bleeding Episode |
52.21; 62.31; 57.85; 45.58; 43.76; 44.56 | — |
| SECONDARY Hemostatic Efficacy Rating for Bleeding Episodes Treated With BAX 855 at Resolution of Bleed |
15; 19; 34; 9; 20; 29 | — |
| SECONDARY Serious Adverse Events (SAEs) Possibly or Probably Related to BAX 855 |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Non-serious Adverse Events Possibly or Probably Related to BAX 855 |
1; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Vital Signs |
1; 0; 0 | — |
| SECONDARY Number of Clinically Significant Changes in Clinical Laboratory Parameters (Hematology, Clinical Chemistry, Lipids) |
1; 0; 1; 1; 0; 1 | — |
| SECONDARY Positive Post-baseline Binding Antibodies to Factor VIII (FVIII), Polyethylene Glycol-Factor VIII (PEG-FVIII), PEG and Chinese Hamster Ovary (CHO) Proteins |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Pharmacokinetics (PK): Area Under the Plasma Concentration Versus Time Curve From 0 to ∞ Hours Post-infusion (AUC0-∞) |
14000; 14400; 14200; 11600; 16600; 14400 | — |
| SECONDARY Pharmacokinetics (PK): Area Under the Plasma Concentration Versus Time Curve From 0 to ∞ Hours Post-infusion Per Dose, (AUC0-∞/Dose) |
— | — |
| SECONDARY Pharmacokinetics (PK): Mean Residence Time (MRT) |
13.3; 14.2; 13.8; 12.5; 11.6; 12.0 | — |
| SECONDARY Pharmacokinetics (PK): Clearance (CL) |
0.0775; 0.1250; 0.1030; 0.0933; 0.1040; 0.0994 | — |
| SECONDARY Pharmacokinetics (PK): Plasma Half-life (T1/2) |
9.24; 9.82; 9.56; 8.68; 8.04; 8.33 | — |
| SECONDARY Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) |
1.02; 1.69; 1.39; 1.14; 1.20; 1.18 | — |
| SECONDARY Pharmacokinetics (PK): Incremental Recovery (IR) |
1.8563; 1.8696; 1.8636; 1.7374; 2.0458; 1.9065 | — |
| SECONDARY Pharmacokinetics (PK): Incremental Recovery (IR) of BAX 855 Over Time - One Stage Clotting Assay |
1.6889; 1.7843; 1.7381; 1.8952; 1.8661; 1.8799 | — |
| SECONDARY Pharmacokinetics (PK): Incremental Recovery (IR) of BAX 855 Over Time - Chromogenic Assay |
1.7675; 1.9334; 1.8532; 1.9273; 1.9960; 1.9635 | — |
Eligibility Criteria
Inclusion Criteria
- Severe hemophilia A (Factor VIII (FVIII) 1.5).
- Participant has severe renal impairment (serum creatinine >1.5 times ULN).
- Participant is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone >10 mg/day, or α-interferon) other than anti-retroviral chemotherapy.
- Participant has current or recent (<30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation.
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- Participant has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the Investigator, would affect participant safety or compliance.
- Participant's legal representative is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
Data sourced from ClinicalTrials.gov (NCT02210091) 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.