Phase 1
Completed N=12
An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B
Source: ClinicalTrials.gov NCT02213250 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jul 2016
Primary outcomePrimary: Maximum Observed Plasma Concentration (Cmax) — 0.3880; 0.4226 IU/milliliter (IU/mL)
Summary
The sample size of 12 male Chinese subjects are based on the CFDA requirement for a China PK study and to support the registration in China.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Maximum Observed Plasma Concentration (Cmax) |
0.3880; 0.4226 | — |
| PRIMARY Area Under the Concentration Time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUClast) |
6.930; 9.707 | — |
| PRIMARY Area Under the Concentration Time Curve From Time 0 to Infinity (AUCinf) |
7.841; 11.66 | — |
| PRIMARY Time to Reach Cmax (Tmax) |
0.500; 0.375 | — |
| PRIMARY Volume of Distribution at Steady State (Vss) |
227.9; 218.8 | — |
| PRIMARY Terminal Phase Rate Constant (Kel) |
0.02509; 0.01781 | — |
| PRIMARY Mean Residence Time (MRT) |
35.78; 51.01 | — |
| PRIMARY Plasma Decay Half-Life (t½) |
27.88; 39.56 | — |
| PRIMARY Systemic Clearance (CL) |
6.378; 4.291 | — |
| PRIMARY Incremental Recovery |
0.7759; 0.8199 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (TEAE), Serious Adverse Events (SAE), and Withdrawals Due to Adverse Events (AE) |
— | — |
| SECONDARY Number of Participants With Abnormal Clinical Laboratory Measurements (Without Regard to Baseline Abnormality) |
12 | — |
| SECONDARY Number of Participants With Vital Signs Post-Dose Data Met Criteria of Potential Clinical Concern (Without Regard to Baseline Abnormality) |
1 | — |
| SECONDARY Number of Participants With Inhibitor Development |
— | — |
| SECONDARY Number of Participants With Allergic Reactions |
— | — |
| SECONDARY Number of Subjects With Thrombogenicity |
— | — |
Eligibility Criteria
Inclusion Criteria
- Male Chinese subjects 6 years or older (weight ≥20kg) with moderate to severe hemophilia B (Factor IX activity ≤2%).
- Subjects should not have received an infusion of any Factor IX products for at least 4 days before the administration of BeneFIX on Day 1.
- Subjects must be in a non-bleeding state before the administration of BeneFIX on Day 1.
Exclusion Criteria
- Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing) disease or clinical findings at Screening.
- Diagnosed with any other bleeding disorder in addition to hemophilia B.
- Current FIX inhibitor or history of FIX inhibitor (defined as > Upper Limit of Normal (ULN) of the reporting lab).
Data sourced from ClinicalTrials.gov (NCT02213250). 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.