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

ConditionVon Willebrand Disease (VWD)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorShanghai RAAS Blood Products Co., Ltd.

About this trial

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in patients with von Willebrand disease.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Hemoglobin < 60 g/L;

Platelet count < 80 x 10^9/L;

Trial design

Design model

Parallel

Treatments tested in this trial

  • SR604

    Drug

    SR604 will be administered as SC injection.

Treatment groups

24 Participants
are divided into 1 treatment group
Group A: Multiple-dose exploratory efficacy trial consists of 4 cohortsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Total annualized bleeding rate (ABR) after treatment

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

Secondary outcomes

1

Annualized spontaneous bleeding rate

Time frame
From baseline, through study completion, an average of 52 weeks
2

Annualized traumatic bleeding rate

Time frame
From baseline, through study completion, an average of 52 weeks
3

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
4

EQ-5D-5L health questionnaire utility value

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

Other outcomes

1

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

Time frame
From baseline, through study completion, an average of 52 weeks
2

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);

Time frame
From baseline, through study completion, an average of 52 weeks
3

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);

Time frame
From baseline, through study completion, an average of 52 weeks
4

Categorization of replacement therapeutic agents prior to investigational product administration, at Week 24 of treatment, and over the total treatment period (including treatment period and extended treatment period).

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

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shanghai RAAS Blood Products Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov
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