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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07129343 | A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD) | PHASE3 | RECRUITING | 20 | — | — | Oct 13, 2025 | Nov 25, 2027 | Jan 8, 2026 | 7 | China |
A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. An SAE is any untoward medical occurrence that at any dose: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event in the opinion of the healthcare provider, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above. TEAEs consist of both serious and non-serious adverse events.
Number of participants with severity of TEAE will be reported.
Number of participants with causality related TEAEs and SAEs will be reported.
Number of participants with thromboembolic events and severe hypersensitivity reactions will be reported.
Number of participants who develop neutralizing antibodies to VWF and FVIII will be reported.
Number of participants who develop total binding antibodies to VWF and FVIII will be reported.
Number of participants with clinically significant abnormalities from baseline values in laboratory parameters per investigator assessment will be reported.
Number of participants with clinically significant abnormalities from baseline values in ECG per investigator assessment will be reported.
Number of participants with clinically significant abnormalities from baseline values in vital sign parameters per investigator assessment will be reported.
| Arm | Type | Description |
|---|---|---|
| All Participants With VWD | EXPERIMENTAL | Participants will receive a single intravenous (IV) dose of VONVENDI at baseline PK assessment. During the 12-month on-demand (OD) treatment period, any bleeding episodes requiring replacement therapy with VWF will be treated with VONVENDI with or without ADVATE. Participants may also receive VONVENDI with or without ADVATE intravenous infusions, when indicated deemed necessary for perioperative bleeding management \[major, minor and oral surgery\]. Participants will receive initial dose of VONVENDI of 40 to 80 IU/kg of body weight. |
| Name | Type | Description |
|---|---|---|
| VONVENDI | BIOLOGICAL | VONVENDI is administered by intravenous injection. |
| ADVATE | BIOLOGICAL | ADVATE is administered by intravenous injection. |
Inclusion Criteria 1. Participant must voluntarily sign an institutional review board (IRB)/independent ethics committee-approved written informed consent form after all relevant aspects of the study have been explained and discussed with the participant. 2. Participant has a documented diagnosis o...