Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02502149 | Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale | PHASE3 | COMPLETED | 24 | — | — | Aug 1, 2015 | Jun 1, 2017 | Dec 19, 2020 | 15 | United States, Australia +1 |
| NCT02083965 | Pharmacokinetics of rFVIIIFc at Two Vial Strengths | PHASE1 | COMPLETED | 19 | — | — | Mar 1, 2014 | May 1, 2015 | Dec 19, 2020 | 8 | United States, Australia +1 |
AUCinf is area under the concentration-time curve from time zero to infinity. Results were summarized overall for 15K rFVIIIFc 1000 IU/vial and 6000 IU/Vial (PK2).
Incremental Recovery is defined as the increase in the circulating FVIII activity in international unit per deciliter (IU/dL) per unit dose administered in international unit per kilogram (IU/kg) (IU/dL per IU/kg). Results were summarized overall for 15K rFVIIIFc 1000 IU/vial and 6000 IU/Vial (PK2).
Area under the plasma concentration versus time curve (AUC) from time zero (pre-dose) to extrapolated infinite time (0 - ∞).
The rise in FVIII activity in IU/dL per unit dose administered in IU/kg (IR, K value), as estimated from the FVIII activity data.
| Arm | Type | Description |
|---|---|---|
| rFVIIIFc (15K scale) 1000 IU vial | EXPERIMENTAL | Single injection of rFVIIIFc (current 2K scale) followed by 2 single injections of rFVIIIFc (15K scale) 1000 IU vial at PK2 and PK3 timepoints. Participants will be on prophylaxis regimen along with treatment for bleeding episodes for 26 weeks of treatment period using the rFVIIIFc (15K scale) 1000 IU vial. |
| rFVIIIFc (15K scale) 6000 IU vial | EXPERIMENTAL | Single injection of rFVIIIFc (current 2K scale) followed by 2 single injections of rFVIIIFc high strength vial (15K scale) at PK2 and PK3 timepoints. Participants will be on prophylaxis regimen along with treatment for bleeding episodes for 26 weeks of treatment period using the rFVIIIFc (15K scale) 6000 IU vial. |
| rFVIIIFc 1000 / 3000 PK Assessment | EXPERIMENTAL | A single intravenous (IV) injection of rFVIIIFc 50 IU/kg at a strength of 1000 IU/vial followed by a single IV injection of rFVIIIFc 50 IU/kg at a strength of 3000 IU/vial. Following the PK assessment, participants will receive either an episodic (on-demand) regimen with doses between 20 and 50 IU/kg based on the severity of the bleeding episode, or 1 of 2 prophylactic regimens: 50 IU/kg every 3 to 5 days or 65 IU/kg weekly. Participants will be allowed to switch from one regimen to another if approved by the Investigator. |
| rFVIIIFc 3000 / 1000 PK Assessment | EXPERIMENTAL | A single IV injection of rFVIIIFc 50 IU/kg at a strength of 3000 IU/vial followed by a single IV injection of rFVIIIFc 50 IU/kg at a strength of 1000 IU/vial. Following the PK assessment, participants will receive either an episodic (on-demand) regimen with doses between 20 and 50 IU/kg based on the severity of the bleeding episode, or 1 of 2 prophylactic regimens: 50 IU/kg every 3 to 5 days or 65 IU/kg weekly. Participants will be allowed to switch from one regimen to another if approved by the Investigator. |
| Name | Type | Description |
|---|---|---|
| rFVIIIFc | BIOLOGICAL | As per arm description |
Key Inclusion Criteria: * Have severe hemophilia A, defined as \<1 IU/dL (\<1%) endogenous FVIII as determined by one-stage clotting assay from the central laboratory at Screening. * Previously treated subject, defined as having at least 150 documented prior exposure days (EDs) to any recombinant a...