Phase 3
Completed N=71
Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A
Source: ClinicalTrials.gov NCT01458106 ↗Enrolled (actual)
71
Serious AEs
7.2%
Results posted
Dec 2014
Primary outcomePrimary: Occurrence of FVIII Inhibitor Development — 0; 0; 0; 0 percentage of participants
Summary
The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Occurrence of FVIII Inhibitor Development |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Annualized Bleeding Rate |
0.00; 2.01 | — |
| SECONDARY Annualized Joint Bleeding Rate (Spontaneous) |
0.00; 0.00 | — |
| SECONDARY Participant Assessment of Response to Injections to Treat a Bleeding Episode |
91.4; 93.5; 65.7; 47.8; 25.7; 45.7 | — |
| SECONDARY Physician's Global Assessment of the Participant's Response to His rFVIIIFc Regimen |
96.5; 89.7; 3.5; 9.1; 0; 1.2 | — |
| SECONDARY Annualized rFVIIIFc Consumption Per Participant |
5331.8; 4973.5; 5562.1; 5092.6 | — |
| SECONDARY Number of Days From Last Treatment Injection to a Spontaneous Bleeding Episode |
2.17; 2.55; 2.16; 2.77 | — |
| 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 |
55.56; 51.35; 56.40; 53.49 | — |
| SECONDARY Maximum Plasma Activity (Cmax; One-stage Activated Partial Thromboplastin Time [aPTT] Clotting Assay) |
95.03; 114.94 | — |
| SECONDARY Maximum Plasma Activity (Cmax; Two-stage Chromogenic Assay) |
94.11; 103.80 | — |
| SECONDARY Elimination Half Life (t1/2; One-stage aPTT Clotting Assay) |
12.277; 13.451 | — |
| SECONDARY Elimination Half Life (t1/2; Two-stage Chromogenic Assay) |
14.268; 15.861 | — |
| SECONDARY Clearance (CL; One-stage aPTT Clotting Assay) |
3.4561; 2.6067 | — |
| SECONDARY Clearance (CL; Two-stage Chromogenic Assay) |
3.8600; 3.0486 | — |
| SECONDARY Volume at Steady State (Vss; One-stage aPTT Clotting Assay) |
57.94; 49.51 | — |
| SECONDARY Volume at Steady State (Vss; Two-stage Chromogenic Assay) |
66.48; 63.15 | — |
| SECONDARY Dose Normalized Area Under the Curve (DNAUC; One-stage aPTT Clotting Assay) |
28.93; 38.37 | — |
| SECONDARY Dose Normalized Area Under the Curve (DNAUC; Two-stage Chromogenic Assay) |
25.90; 32.80 | — |
| SECONDARY Mean Residence Time (MRT; One-stage aPTT Clotting Assay) |
16.762; 18.999 | — |
| SECONDARY Mean Residence Time (MRT; Two-stage Chromogenic Assay) |
17.220; 20.708 | — |
| SECONDARY Incremental Recovery (IR; One-stage aPTT Clotting Assay) |
1.901; 2.299 | — |
| SECONDARY Incremental Recovery (IR; Two-stage Chromogenic Assay) |
1.882; 2.076 | — |
| SECONDARY Time at Maximum Activity (Tmax; One-stage aPTT Clotting Assay) |
0.6987; 0.7257 | — |
| SECONDARY Time at Maximum Activity (Tmax; Two-stage Chromogenic Assay) |
0.7313; 0.6334 | — |
| SECONDARY Lambda Z (One-stage aPTT Clotting Assay) |
0.05644; 0.05158 | — |
| SECONDARY Lambda Z (Two-stage Chromogenic Assay) |
0.04848; 0.04367 | — |
| SECONDARY Volume at Terminal Phase (Vz; One-stage aPTT Clotting Assay) |
61.22; 50.58 | — |
| SECONDARY Volume at Terminal Phase (Vz; Two-stage Chromogenic Assay) |
79.48; 69.75 | — |
| SECONDARY Area Under the Curve to the Last Measurable Timepoint (AUClast; One-stage aPTT Clotting Assay) |
1410.4; 1823.4 | — |
| SECONDARY Area Under the Curve to the Last Measurable Timepoint (AUClast; Two-stage Chromogenic Assay) |
1250.1; 1540.4 | — |
| SECONDARY Area Under the Curve to Infinity (AUCinf; One-stage aPTT Clotting Assay) |
1446.5; 1918.5 | — |
| SECONDARY Area Under the Curve to Infinity (AUCinf; Two-stage Chromogenic Assay) |
1294.7; 1640.0 | — |
| SECONDARY Percentage of AUCinf Extrapolated From the Last Data Point to Infinity (%AUCext; One-stage aPTT Clotting Assay) |
1.8421; 2.7777 | — |
| SECONDARY Percentage of AUCinf Extrapolated From the Last Data Point to Infinity (%AUCext; Two-stage Chromogenic Assay) |
2.7530; 3.9476 | — |
Eligibility Criteria
Key Inclusion Criteria
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII
- Male <12 years of age and weight ≥13 kg
- History of at least 50 documented prior exposure days to FVIII
- No current, or history of, inhibitor development to FVIII
Key Exclusion Criteria
- Other coagulation disorders in addition to Hemophilia A
- History of anaphylaxis associated with any FVIII or IV immunoglobulin administration
NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT01458106). 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.