von Willebrand Disease type 2B
Conditions
Interventions
Product Name: ARC1779
Product Code: ARC1779
Pharmaceutical Form: Solution for infusion
Sponsors
Medical University of Vienna, Dept. of Clinical Pharmacology
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the effect of ARC1779 Injection infusion on platelet count in VWD-2B patients who have chronic thrombocytopenia; ;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 To evaluate the safety and tolerability of ARC1779 Injection in VWD-2B patients. ;Primary end point(s): platelet counts | — |
Countries
Austria
Outcome results
None listed