A Study of the Pharmacodynamics, Pharmacokinetics, and Safety & Efficacy of ARC1779 Injection in Patients with von Willebrand Disease Type 2B - ARC1779 for VWD-2B

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

•Male or female patients;
•=18 to = 75 years of age;
•Diagnosis of VWD-2B according to national expert guidelines for the USA [1] and Europe [2] based on medical history and findings from a matrix of laboratory assays which may include: platelet count, concentration of VWF antigen (VWF:Ag), VWF ristocetin cofactor activity (VWF:RiCo), Factor VIII (FVIII) activity, ristocetin-induced platelet aggregation (RIPA), platelet function analyzer (PFA-100®) closure time, cutaneous bleeding time (CBT), VWF multimer test, VWF: platelet-binding (VWF:PB) activity, etc.;
•Chronic thrombocytopenia, defined as a platelet count <100 per nL on at least 2 occasions within the 30 days preceding enrollment;
•Female patients of reproductive age must be enrolled within 1 to 7 days of the cessation of preceding menses;
•Female patients must be non-pregnant and willing to use effective, redundant methods of contraception (i.e., for both self and male partner) throughout the study and for at least 30 days after discontinuation of study drug treatment;
•Male patients must agree to use a medically acceptable contraceptive (abstinence or use of a condom with spermicide) throughout the study and for at least 30 days after discontinuation of study drug treatment;
•All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Patients with a possible co-existing or alternative hematologic diagnosis which can account for the laboratory findings of thrombocytopenia, etc.;
•Co-existing conditions which are associated with an increased risk of intraparenchymal bleeding, e.g. recent major trauma or surgery
•Pregnancy

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the effect of ARC1779 Injection infusion on platelet count in VWD-2B patients who have chronic thrombocytopenia;<br>;Secondary Objective: To determine the effect of ARC1779 Injection infusion on von Willebrand Factor Antigen, Ristocetin Cofactor Activity, Factor VIII, Multimers, activated partial thromboplastin time, platelet aggregation, PFA-100 and other coagulation tests <br>To evaluate the safety and tolerability of ARC1779 Injection in VWD-2B patients.<br>;Primary end point(s): platelet counts

Secondary Outcome Measures