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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02234310 | Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B | PHASE3 | COMPLETED | 33 | — | — | Nov 13, 2014 | Aug 20, 2019 | Mar 25, 2022 | 27 | United States, Australia +9 |
| NCT00716716 | Phase I/IIa Study of FIXFc in Hemophilia B Patients | PHASE1 | COMPLETED | 10 | — | — | Apr 1, 2008 | Oct 1, 2009 | Dec 19, 2020 | 6 | United States |
Development of an inhibitor was defined as an inhibitor test result of \>= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of \>=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
| Arm | Type | Description |
|---|---|---|
| Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | EXPERIMENTAL | Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (\>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX (FIX) levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR. |
| rFIXFc | EXPERIMENTAL | Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg |
| Name | Type | Description |
|---|---|---|
| rFIXFc | BIOLOGICAL | Adjustments to the dose and interval of rFIXFc was made in this study based on investigator discretion using available pharmacokinetic (PK) data, subsequent FIX trough and peak levels, level of physical activity, and bleeding pattern, in accordance with local standards of care for a prophylactic regimen. There was an option to start study dosing as episodic treatment (on-demand). |
Key Inclusion Criteria: * Weight \>=3.5 kilogram at the time of informed consent. * Severe hemophilia B was defined as less than or equal to (\<=)2 International Units per deciliter (IU/dL) (\<=2 percent \[%\]) endogenous FIX documented in the medical record or as tested during the Screening Period...