Von Willebrand Disease (VWD)
Conditions
Brief summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in patients with von Willebrand disease.
Interventions
DRUGSR604
SR604 will be administered as SC injection.
Sponsors
Shanghai RAAS Blood Products Co., Ltd.
Study design
Allocation
NA
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Patients must meet ALL of the following inclusion criteria to be enrolled: 1. Age \>= 18 years and \<= 65 years at the time of signing informed consent, regardless of sex; 2. At screening, patients with a confirmed diagnosis of von Willebrand disease (VWD) with documented evidence and a defined VWD subtype; 3. At least 4 new bleeding episodes within 6 months prior to screening; 4. No active bleeding symptoms prior to the first dose; 5. The subject or impartial witness fully understands and is able to comply with the protocol requirements, is willing to complete the study as planned, and voluntarily agrees to provide biological samples for testing as required by the protocol; is able to understand the procedures and methods of this clinical trial, provides voluntary participation after full informed consent, and personally signs the informed consent form.
Exclusion criteria
* Patients meeting ANY of the following
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Total annualized bleeding rate (ABR) after treatment | From baseline, through study completion, an average of 52 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized spontaneous bleeding rate | From baseline, through study completion, an average of 52 weeks | — |
| Annualized traumatic bleeding rate | From baseline, through study completion, an average of 52 weeks | — |
| Overall annualized bleeding rate, annualized spontaneous bleeding rate, and annualized traumatic bleeding rate | From baseline, through study completion, an average of 52 weeks | — |
| EQ-5D-5L health questionnaire utility value | From baseline, through study completion, an average of 52 weeks | — |
| Change in EQ-VAS score from baseline | From baseline, through study completion, an average of 52 weeks | — |
| PK parameters after first dose:Peak Plasma Concentration (Cmax) | Day1 | — |
| Pharmacokinetic parameters after multiple doses: Peak Plasma Concentration (Cmax) | From baseline, through study completion, an average of 52 weeks | — |
| Incidence of Adverse Events (AEs) | From baseline, through study completion, an average of 52 weeks | Number of participants experiencing at least one AE |
| PK parameters after first dose:Time to Peak Plasma Concentration (Tmax) | Day1 | — |
| Safety: Number and incidence of patients with anti-drug antibodies (ADA) | From baseline, through study completion, an average of 52 weeks | — |
| Pharmacokinetic parameters after multiple doses: Time to Peak Plasma Concentration (Tmax) | From baseline, through study completion, an average of 52 weeks | — |
| Incidence of Serious Adverse Events (SAEs) | From baseline, through study completion, an average of 52 weeks | Number of participants experiencing at least one SAE |
| Incidence of Adverse Events of Special Interest (AESIs) | From baseline, through study completion, an average of 52 weeks | Number of participants experiencing at least one AESI |
| Pharmacodynamic indicators:protein C | From baseline, through study completion, an average of 52 weeks | — |
| Pharmacodynamic indicators:prothrombin time (PT) | From baseline, through study completion, an average of 52 weeks | — |
| Pharmacokinetic parameters after multiple doses:Time to Peak Plasma Concentration (Tmax) | From baseline, through study completion, an average of 52 weeks | — |
| Pharmacokinetic parameters after multiple doses: Area Under the Concentration-Time Curve from Zero to Last Quantifiable Time Point (AUC0-t) | From baseline, through study completion, an average of 52 weeks | — |
Countries
China
Contacts
CONTACTResearch and Development
Outcome results
None listed