Von Willebrand Disease (VWD)Phase 2NCT07640893
A Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of SR604 Injection in Patients With Von Willebrand Disease
A Phase 2 study testing SR604 in people with Von Willebrand Disease (VWD), run by Shanghai RAAS Blood Products Co., Ltd. at 9 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Von Willebrand Disease (VWD)
- What is being tested?
- SR604
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 9 sitesChina
- Study dates
- 26 Nov 2025to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must meet ALL of the following inclusion criteria to be enrolled:
- Age >= 18 years and <= 65 years at the time of signing informed consent, regardless of sex;
- At screening, patients with a confirmed diagnosis of von Willebrand disease (VWD) with documented evidence and a defined VWD subtype;
- At least 4 new bleeding episodes within 6 months prior to screening;
- No active bleeding symptoms prior to the first dose;
- 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.
Who cannot join
- Patients meeting ANY of the following exclusion criteria will not be enrolled:
- Known history of hypersensitivity to the investigational drug formulation or any of its components;
- Intolerance to subcutaneous injection or presence of other local skin abnormalities or dermatological conditions that may affect drug administration and safety assessment;
- Meeting any of the following criteria at screening:
- Hemoglobin < 60 g/L;
- Platelet count < 80 x 10^9/L;
Show all 26Show fewer
- Hepatic or renal dysfunction: Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >= 2.5 x upper limit of normal (ULN), or total bilirubin >= 1.5 x ULN; or serum creatinine (Cr) >= 1.5 x ULN;
- Positive for anti-human immunodeficiency virus (HIV) antibody;
- Presence of any bleeding disorder other than von Willebrand disease [hemophilia A or B, congenital coagulation factor VII deficiency, acquired von Willebrand disease (AVWS), platelet-type VWD, inherited platelet disorders, etc.]; or significantly abnormal coagulation parameters due to diseases other than von Willebrand disease (e.g., platelet disorders, vitamin K deficiency, etc.);
- Presence of protein C deficiency or protein S deficiency;
- History of thrombosis or family history of thrombosis prior to signing informed consent or currently, or history of thrombophilia;
- Severe bleeding due to VWD within 2 years prior to screening, such as intracranial hemorrhage, esophageal variceal bleeding, etc.;
- Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association class >= III), severe arrhythmia (QTc interval > 500 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic blood pressure >= 160 mmHg or diastolic blood pressure >= 100 mmHg), etc.;
- Female patients with menstrual abnormalities due to organic gynecological diseases (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);
- Previous or current life-threatening malignant neoplasms or end-stage liver disease;
- Use of DDAVP or plasma-derived VWF-containing factor VIII concentrate, plasma-derived/recombinant VWF preparations, or antifibrinolytic therapy within 1 week prior to the first dose;
- Use of antithrombotic agents within 1 week prior to the first dose;
- Receipt of fresh blood/plasma or cryoprecipitate therapy within 2 weeks prior to the first dose;
- Receipt of vaccination within 1 month prior to the first dose or planned vaccination during the study period;
- Major surgery (major surgery defined as Grade III and IV surgeries) within 1 month prior to the first dose, or planned surgery during the study period;
- Enrollment in other clinical trials within 1 month prior to the first dose;
- History of drug abuse or alcohol dependence (alcohol dependence criteria: long-term drinking history exceeding 5 years, equivalent ethanol intake >= 40 g/day, or heavy drinking within 2 weeks, equivalent ethanol intake > 80 g/day. Ethanol amount (g) conversion formula = alcohol consumption (mL) x alcohol content (%) x 0.8);
- Presence of psychiatric disease or significant mental disorder, or other reasons resulting in incapacity or lack of cognitive ability;
- Plans for procreation or sperm donation throughout the study period up to 3 months after the last dose, or unwillingness to use effective physical contraceptive measures (e.g., condoms);
- Presence of clinically significant disease or other reasons rendering the patient unsuitable for clinical trial participation in the investigator's opinion (e.g., patient unlikely to benefit from the clinical trial);
- Patients whom the investigator considers to have poor compliance, rendering efficacy evaluation difficult or likelihood of completing the planned treatment course and follow-up low.
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
China9 sites, 9 recruiting
- Recruiting
Xiangya Hospital of Central South University
Changsha
- Recruiting
The First Affiliated Hospital of University of Science and Technology of China
Hefei
- Recruiting
Jinan Central Hospital
Jinan
- Recruiting
The First Affiliated Hospital of Guangxi Medical University
Nanning
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou
- Recruiting
The Second Hospital of Shanxi Medical University
Taiyuan
- Recruiting
North China University of Science and Technology Affiliated Hospital
Tangshan
- Recruiting
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin
- Recruiting
Henan Provincial People's Hospital
Zhengzhou
About this study
Official title: A Multi-Dose, Randomized, Multicenter Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Profile of SR604 Injection in Patients With Von Willebrand Disease
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in patients with von Willebrand disease.
- Conditions
- Von Willebrand Disease (VWD)
- Being tested
- SR604
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SR604Drug
SR604 will be administered as SC injection.
| Study group | Role | What they receive |
|---|---|---|
| Multiple-dose exploratory efficacy trial consists of 4 cohorts | Experimental | Participants with Von Willebrand Disease will receive SR604 dose 1 as multiple SC injections every 4-weeks, or dose 2 as multiple SC injections every 4-weeks/6-weeks/8-weeks. Receives: Drug: SR604 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total annualized bleeding rate (ABR) after treatment
Measured: From baseline, through study completion, an average of 52 weeks
Secondary outcomes (17)
Annualized spontaneous bleeding rate
Measured: From baseline, through study completion, an average of 52 weeks
Annualized traumatic bleeding rate
Measured: From baseline, through study completion, an average of 52 weeks
Overall annualized bleeding rate, annualized spontaneous bleeding rate, and annualized traumatic bleeding rate
Measured: From baseline, through study completion, an average of 52 weeks
EQ-5D-5L health questionnaire utility value
Measured: From baseline, through study completion, an average of 52 weeks
Change in EQ-VAS score from baseline
Measured: From baseline, through study completion, an average of 52 weeks
PK parameters after first dose:Peak Plasma Concentration (Cmax)
Measured: Day1
Pharmacokinetic parameters after multiple doses: Peak Plasma Concentration (Cmax)
Measured: From baseline, through study completion, an average of 52 weeks
Incidence of Adverse Events (AEs)
Number of participants experiencing at least one AE
Measured: From baseline, through study completion, an average of 52 weeks
PK parameters after first dose:Time to Peak Plasma Concentration (Tmax)
Measured: Day1
Safety: Number and incidence of patients with anti-drug antibodies (ADA)
Measured: From baseline, through study completion, an average of 52 weeks
Pharmacokinetic parameters after multiple doses: Time to Peak Plasma Concentration (Tmax)
Measured: From baseline, through study completion, an average of 52 weeks
Incidence of Serious Adverse Events (SAEs)
Number of participants experiencing at least one SAE
Measured: From baseline, through study completion, an average of 52 weeks
Incidence of Adverse Events of Special Interest (AESIs)
Number of participants experiencing at least one AESI
Measured: From baseline, through study completion, an average of 52 weeks
Pharmacodynamic indicators:protein C
Measured: From baseline, through study completion, an average of 52 weeks
Pharmacodynamic indicators:prothrombin time (PT)
Measured: From baseline, through study completion, an average of 52 weeks
Pharmacokinetic parameters after multiple doses:Time to Peak Plasma Concentration (Tmax)
Measured: 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)
Measured: From baseline, through study completion, an average of 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2026
- Study start26 Nov 2025
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai RAAS Blood Products Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07640893
- Sponsor's protocol ID
- LS-SR604-VWD-II01
- Status last verified
- May 2026
- First submitted
- 1 Jun 2026
- Last updated
- 11 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Dose, Randomized, Multicenter Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Profile of SR604 Injection in Patients With Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT07640893
- Acronym / sponsor ID
- LS-SR604-VWD-II01
- Conditions
- Von Willebrand Disease (VWD)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Shanghai RAAS Blood Products Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 26 Nov 2025 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 11 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in patients with von Willebrand disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Multiple-dose exploratory efficacy trial consists of 4 cohorts | Experimental | Drug: SR604Participants with Von Willebrand Disease will receive SR604 dose 1 as multiple SC injections every 4-weeks, or dose 2 as multiple SC injections every 4-weeks/6-weeks/8-weeks. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients must meet ALL of the following inclusion criteria to be enrolled:
Key exclusion criteria (26 listed)
- Patients meeting ANY of the following exclusion criteria will not be enrolled:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SR604SR604 will be administered as SC injection. | Drug | Multiple-dose exploratory efficacy trial consists of 4 cohorts |
Primary outcome
Total annualized bleeding rate (ABR) after treatment
Time frame: From baseline, through study completion, an average of 52 weeks
17 secondary outcomes
Annualized spontaneous bleeding rate
Time frame: From baseline, through study completion, an average of 52 weeks
Annualized traumatic bleeding rate
Time frame: From baseline, through study completion, an average of 52 weeks
Overall annualized bleeding rate, annualized spontaneous bleeding rate, and annualized traumatic bleeding rate
Time frame: From baseline, through study completion, an average of 52 weeks
EQ-5D-5L health questionnaire utility value
Time frame: From baseline, through study completion, an average of 52 weeks
Change in EQ-VAS score from baseline
Time frame: From baseline, through study completion, an average of 52 weeks
PK parameters after first dose:Peak Plasma Concentration (Cmax)
Time frame: Day1
Pharmacokinetic parameters after multiple doses: Peak Plasma Concentration (Cmax)
Time frame: From baseline, through study completion, an average of 52 weeks
Incidence of Adverse Events (AEs)
Time frame: From baseline, through study completion, an average of 52 weeks
PK parameters after first dose:Time to Peak Plasma Concentration (Tmax)
Time frame: Day1
Safety: Number and incidence of patients with anti-drug antibodies (ADA)
Time frame: From baseline, through study completion, an average of 52 weeks
Pharmacokinetic parameters after multiple doses: Time to Peak Plasma Concentration (Tmax)
Time frame: From baseline, through study completion, an average of 52 weeks
Incidence of Serious Adverse Events (SAEs)
Time frame: From baseline, through study completion, an average of 52 weeks
Incidence of Adverse Events of Special Interest (AESIs)
Time frame: From baseline, through study completion, an average of 52 weeks
Pharmacodynamic indicators:protein C
Time frame: From baseline, through study completion, an average of 52 weeks
Pharmacodynamic indicators:prothrombin time (PT)
Time frame: From baseline, through study completion, an average of 52 weeks
Pharmacokinetic parameters after multiple doses:Time to Peak Plasma Concentration (Tmax)
Time frame: 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)
Time frame: From baseline, through study completion, an average of 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SR604 in people with Von Willebrand Disease (VWD), run by Shanghai RAAS Blood Products Co., Ltd. at 9 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Research and Development (862122130888, hanyu@raas-corp.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total annualized bleeding rate (ABR) after treatment (time frame: From baseline, through study completion, an average of 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026.