A Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of SR604 Injection in Patients With Von Willebrand Disease

NCT07640893 | Recruiting Phase 2

• 1 other identifier: LS-SR604-VWD-II01
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 9

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.

Conditions

Von Willebrand Disease (VWD)

Outcome Measures

Primary Outcomes (1)

• Total annualized bleeding rate (ABR) after treatment

  From baseline, through study completion, an average of 52 weeks

Secondary Outcomes (17)

• 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

Other Outcomes (4)

• Pharmacodynamic indicators:Protac-APTT (Protac-induced protein C-activated APTT assay)

  From baseline, through study completion, an average of 52 weeks

• Change from baseline in PBAC score at Week 24 of treatment and over the total treatment period (including treatment period and extended treatment period) (females with menstruation only);

  From baseline, through study completion, an average of 52 weeks

• Annualized menorrhagia bleeding rate at Week 24 of treatment and over the total treatment period (including treatment period and extended treatment period) (females with menstruation only);

  From baseline, through study completion, an average of 52 weeks

Study Arms (1)

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.

Drug: SR604

Interventions

SR604 DRUG

SR604 will be administered as SC injection.

Multiple-dose exploratory efficacy trial consists of 4 cohorts

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;
• 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;

Sponsors & Collaborators

• Shanghai RAAS Blood Products Co., Ltd.: lead

Study Sites (9)

Xiangya Hospital of Central South University

Changsha, China

RECRUITING

The First Affiliated Hospital of University of Science and Technology of China

Hefei, China

RECRUITING

Jinan Central Hospital

Jinan, China

RECRUITING

The First Affiliated Hospital of Guangxi Medical University

Nanning, China

RECRUITING

The First Affiliated Hospital of Soochow University

Suzhou, China

RECRUITING

The Second Hospital of Shanxi Medical University

Taiyuan, China

RECRUITING

North China University of Science and Technology Affiliated Hospital

Tangshan, China

RECRUITING

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, China

RECRUITING

Henan Provincial People's Hospital

Zhengzhou, China

RECRUITING

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Research and Development

CONTACT

862122130888; hanyu@raas-corp.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2026
• First Posted: June 11, 2026
• Study Start: November 26, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: June 11, 2026

Record last verified: 2026-05

Locations

CN (9)