Type III von Willebrand disease
Conditions
Interventions
emicizumab: To administer emicizumab subcutaneously at the designated dose.
Sponsors
Kanwal John Pasi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Signed Informed Consent Form (signed by participant's legally authorized representative for participants who have not attained the age of maturity) Signed Assent Form when appropriate, as determined by the prospective participant's age and individual site and country standards Confirmed diagnosis of Type III VWD, based on medical records (all of the below samples are required to be from one setting)
Exclusion criteria
Exclusion criteria: Pregnant or breastfeeding, or intending to become pregnant during the study or within the time frame in which contraception is required. Participants of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study treatment. Receipt of any of the following: - Emicizumab in a prior investigational study or during the observation period in Study WP45335 (Arm C) - Emicizumab within 24 weeks prior to enrollment(Arm A and Arm B) - An investigational drug to treat or reduce the risk of VWD-related bleeds within 5 half-lives of last drug administration - Any future planned or concurrent non-VWD-related investigational drug, within last 30 days or 5 half-lives, whichever is shorter Any inherited or acquired bleeding disorder other than congenital Type III VWD
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy, confirmatory To evaluate efficacy of emicizumab by number of treated bleeds over time. | — |
Secondary
| Measure | Time frame |
|---|---|
| safety, efficacy, exploratory, other To evaluate efficacy and safety of Emicizumab by following: - Number of treated bleeds over time - Number of treated spontaneous bleeds over time - Number of treated joint bleeds over time - Incidence and severity of adverse events - Incidence and severity of thromboembolic events with severity - Incidence and severity of thrombotic microangiopathy events - Changes from baseline in physical examination findings, vital signs, and ECG parameters | — |
Countries
Belgium, Canada, Colombia, France, Germany, Italy, Japan, Netherlands, Poland, South Africa, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactClinical trials information
Chugai Pharmaceutical Co., Ltd.
Outcome results
None listed