patients with type 2B Von Willebrand Disease with thrombocytopenia MedDRA version: 20.0 Level: PT Classification code 10047715 Term: Von Willebrand's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: rondoraptivon pegol
Product Code: BT200
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Rondoraptivon pegol
Current Sponsor code: BT200
Other descriptive name: Poly(oxy-
Sponsors
Medical University of Vienna, Department of Clinical Pharmacology
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Type 2B VWD with thrombocytopenia and a recent bleeding history 2. repeated thrombocytopenia in medical history 3. =18 years old 4. Able to comprehend and to give informed consent 5. Able to cooperate with the Investigator, to comply with the requirements of the study, and to complete the full sequence of protocol-related procedures 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 5 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1. Clinically significant medical history or ongoing chronic illness that would jeopardise the safety of the patient or compromise the quality of the data derived from his/her participation in this study 2. History of significant drug allergy or anaphylactic reactions 3. Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the patient to be able to comply fully with study procedures 4. Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the patient’s welfare or the integrity of the study’s results 5. Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 elimination half-lives (whichever is longer) prior to treatment start
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To examine the effects of BT200 on platelet count, VWF indices, FVIII and bleeding in type 2B VWD ;Secondary Objective: The secondary objective of this study is to assess the pharmacokinetic (PK)/pharmacodynamic (PD) relationship for BT200 in patients with type 2B VWD. To further evaluate the safety and tolerability of BT200 in patients with type 2B VWD. ;Primary end point(s): Co-Primary endpoints: Platelet counts, number of monthly bleedings events ;Timepoint(s) of evaluation of this end point: blood will be collected on day 1 (=baseline), day 29, day 57, day 85 and the platelet counts will be quantified. bleeding assessments will be done during the active study period of approximately 3 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacokinetics: • BT200 concentrations (and derived PK parameters) • Half-life of the substituted Factor VIII product used with and without BT200 (pop-PK-sub-study) Pharmacodynamics: • VWF antigen (VWF Ag) • VWF:ristocetin co-factor assay (VWF:RCo) • VWF activity (VWF:GpIbM assay) • VWF collagen binding assay (VWF:CBA) • Enzyme-linked immunosorbent assay (ELISA) for unbound VWF A1 domain (REAADS®) • Whole blood ristocetin induced platelet aggregation (Multiplate®) • Collagen Adenosine Diphosphate closure times measured by the platelet function analyser (PFA-100®) ;Timepoint(s) of evaluation of this end point: blood will be collected on day 1 (=baseline), day 29, day 57, day 85 and the platelet counts will be quantified. bleeding assessments will be done during the active study period of approximately 3 months | — |
Countries
Austria
Contacts
Public ContactOffice of the Department
Medical University of Vienna, Department of Clinical Pharmacology
Outcome results
None listed