Phase 3
N=30
Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01440946 ↗Enrolled (actual)
30
Serious AEs
13.3%
Results posted
Jun 2015
Primary outcome: Primary: Occurence of Factor IX (FIX) Inhibitor Development — 0; 0; 0; 0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rFIXFc (Drug); FIX (Drug)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- Bioverativ Therapeutics Inc.
- Primary completion
- Nov 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Occurence of Factor IX (FIX) Inhibitor Development |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Annualized Bleeding Rate |
1.09; 2.13 | — |
| SECONDARY Annualized Joint Bleeding Rate (Spontaneous) |
0.00; 0.00 | — |
| SECONDARY Participant Assessment of Response to Injections to Treat a Bleeding Episode |
89.5; 88.2; 52.6; 41.2; 36.8; 47.1 | — |
| SECONDARY Physician's Global Assessment of the Participant's Response to His rFIXFc Regimen |
85.4; 89.8; 14.6; 10.2; 0; 0 | — |
| SECONDARY Annualized rFIXFc Consumption by Type of Injection |
3041.5; 3185.6; 147.9; 293.8; 29.2; 16.9 | — |
| SECONDARY Number of Days From the Last Prophylaxis Injection to a Spontaneous Bleeding Episode |
3.97; 5.55; 4.12; 5.52 | — |
| SECONDARY Number of Injections Required for Resolution of a Bleeding Episode |
1.0; 1.0; 1.0; 1.0 | — |
| SECONDARY Total Dose Required for Resolution of a Bleeding Episode |
65.37; 89.77; 70.22; 52.22 | — |
| SECONDARY Maximum Plasma Activity (Cmax; One-stage Activated Partial Thromboplastin Time [aPTT] Clotting Assay) |
29.78; 35.84 | — |
| SECONDARY Terminal Half Life (t1/2; One-stage aPTT Clotting Assay) |
66.49; 70.34 | — |
| SECONDARY Clearance (CL; One-stage aPTT Clotting Assay) |
4.365; 3.505 | — |
| SECONDARY Volume of Distribution at Steady State (Vss; One-stage aPTT Clotting Assay) |
365.1; 289.0 | — |
| SECONDARY Dose Normalized Area Under the Curve (DNAUC; One-stage aPTT Clotting Assay) |
22.71; 28.53 | — |
| SECONDARY Mean Residence Time (MRT; One-stage aPTT Clotting Assay) |
83.65; 82.46 | — |
| SECONDARY Incremental Recovery (IR; One-stage aPTT Clotting Assay) |
0.5898; 0.7170 | — |
Summary
The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFIXFc; to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes
Eligibility Criteria
Key Inclusion Criteria
- Severe hemophilia B defined as ≤ 2 IU/dl (≤ 2%) endogenous FIX
- Male < 12 years and weight ≥ 13 kg
- History of at least 50 documented prior exposure days to FIX
- No history of, or currently detectable, inhibitor
Key Exclusion Criteria
- Other coagulation disorders in addition to Hemophilia B
- History of anaphylaxis associated with any FIX or IV immunoglobulin administration
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT01440946). 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.