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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02932618 | A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) | PHASE3 | COMPLETED | 47 | — | — | Nov 6, 2017 | Apr 17, 2026 | May 15, 2026 | 46 | United States, Austria +11 |
Treatment success for vonicog alfa-treated nonsurgical bleeding episodes (using a 4-point scale: Excellent, Good, Moderate, None).
| Arm | Type | Description |
|---|---|---|
| On-demand Treatment | EXPERIMENTAL | Participants will receive vonicog alfa (recombinant von Willebrand factor \[rVWF\]) treatment for non-surgical bleeding episodes over a 12 to 18-month period. |
| Elective Surgery | EXPERIMENTAL | 12-24 hours prior to surgery and within 3 hours of surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing. |
| Emergency Surgery | EXPERIMENTAL | Within 3 hours prior to surgery. Minor surgery: infuse every 12-24 hours for at least 48 hours based on post-operative dosing. Oral Surgery: infuse at least once within first 8-12 hours post-surgery based on post-operative dosing. Major Surgery: infuse every 12-24 hours for at least first 96 hours post-surgery based on post-operative dosing. |
| Name | Type | Description |
|---|---|---|
| Vonicog alfa | BIOLOGICAL | Lyophilized powder and solvent to prepare solution for injection. |
| Antihemophilic Factor (Recombinant) | BIOLOGICAL | Packaged in single boxes with 2 glass vials, with one vial containing the lyophilized ADVATE and the second vial containing the diluent. |
Inclusion Criteria: * Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]): * Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or * Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diag...