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Also known as Recombinant von Willebrand Factor (rVWF)
Recombinant von Willebrand Factor · 2 trials · 1 indication
Hemostatic efficacy will be rated on a scale of excellent - good - moderate - none. Excellent: Intra-, and postoperative hemostasis achieved with rVWF with our without ADVATE was as good or better than that expected for the type of surgical procedure performed in a hemostatically normal subject. Good: Intra-, and postoperative hemostasis achieved with rVWF with or without ADVATE was probably as good as that expected for the type of surgical procedure performed in a hemostatically normal subject. Moderate: Intra-, and postoperative hemostasis with rVWF with or without ADVATE was clearly less than optimal for the type of procedure performed but was maintained without the need to change the rVWF concentrate. None: Participant experienced uncontrolled bleeding that was the result of inadequate therapeutic response despite proper dosing, necessitating a change of rVWF concentrate.
Treatment success was defined as the extent of control of bleeding episodes (BEs) using a mean efficacy rating score of \<2.5 for a participant's BEs treated with study product (recombinant von Willebrand Factor \[rVWF\] with or without recombinant factor VIII \[rFVIII\]) during the study period. Scores used: Excellent = 1 - actual infusions ≤ estimated number of infusions required to treat BE; no additional VWF required (all BEs); Good = 2 - \>1-2 infusions (minor/moderate BEs) or \<1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); Moderate = 3 ≥ 3 infusions (minor/moderate BEs) or ≥ 1.5 infusions (major BEs) greater than estimated required to control BE; no additional VWF required (all BEs); None = 4 - severe uncontrolled bleeding or intensity of bleeding not changed; additional VWF required. Included participants with available primary efficacy rating (prospective-excluding gastrointestinal bleeds) in the Full Analysis Set.
| Arm | Type | Description |
|---|---|---|
| Recombinant von Willebrand Factor (rVWF) | EXPERIMENTAL | Surgery participants treated with Recombinant von Willebrand Factor (rVWF) |
| PK 80 Arm (minimum of 22 subjects with severe VWD) | EXPERIMENTAL | PK assessment (80 IU/kg rVWF) + 12-month treatment period |
| PK 50 Arm (14 subjects with type 3 VWD) | EXPERIMENTAL | Two single-blinded PK assessments (50 IU/kg rVWF + rFVIII/placebo) + 12-month treatment period |
| PK 50 Only Arm (minimum of 7 subjects with type 3 VWD) | EXPERIMENTAL | PK assessment (50 IU/kg rVWF) only, no treatment of bleeding episodes |
| Treatment Only (up to 7 subjects independent of VWD subtype) | EXPERIMENTAL | Treatment of bleeding episodes for a total of 12 months |
| Name | Type | Description |
|---|---|---|
| Recombinant von Willebrand Factor (rVWF) | BIOLOGICAL | rVWF will be administered by intravenous bolus infusion. Participants planned for major surgery will undergo a baseline pharmacokinetic assessment prior to surgery. The peri- and postoperative substitution regimen will be individualized according to the PK results, intensity and duration of the hemostatic challenge, and the institution´s standard of care. |
| Placebo | DRUG | Syringe supplied with physiologic saline solution for infusion |
| Recombinant factor VIIII (rFVIII) | BIOLOGICAL | Intravenous administration |
Inclusion Criteria: * Diagnosis of severe von Willebrand disease (VWD) as listed below and elective surgical procedure planned 1. Type 1 (Von Willebrand factor : Ristocetin cofactor activity (VWF:RCo) \<20 IU/dL), or 2. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genoty...
Top 4 of 5 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 4 | PHASE3 | Vonvendi, ADVATE |
| Incyte Corporation | INCY | 2 | PHASE3 | VGA039 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE2 | ALN-6400 |
| Hemab Therapeutics Holdings, Inc. | COAG | 2 | PHASE1 | HMB-002 |
Recombinant von Willebrand Factor is used to treat von Willebrand disease, a bleeding disorder. It is studied in patients with severe von Willebrand disease, including those undergoing surgery and those experiencing bleeding episodes. It is a recombinant form of the von Willebrand factor protein.
Recombinant von Willebrand Factor targets factor IX (F9). It is an exogenous protein, meaning it is a recombinant version of a naturally occurring protein. By targeting F9, it is designed to support blood clotting in patients with von Willebrand disease.
Recombinant von Willebrand Factor is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the company responsible for its clinical development.
Recombinant von Willebrand Factor is in Phase 3 clinical development. It is an investigational product and has not been approved by the FDA. Two Phase 3 trials have been completed.
Recombinant von Willebrand Factor has been studied in two completed Phase 3 trials: NCT02283268, which evaluated it in subjects with severe von Willebrand disease undergoing surgery, and NCT01410227, which assessed its pharmacokinetics, safety, and efficacy in treating bleeding episodes in von Willebrand disease.
Yes, Recombinant von Willebrand Factor is also known as rVWF, which stands for recombinant von Willebrand factor. Both names refer to the same investigational product developed by Takeda.