None listed
Conditions
Interventions
Product Name: BT200
Pharmaceutical Form: Solution for injection
INN or Proposed INN: BT200
CAS Number: not assigned
Other descriptive name: BT200
Concentration unit: mg/ml milligram(s)/millilitre
Conc
Sponsors
Medical University of Vienna
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible for this study, subjects must meet all of the following inclusion criteria: 1. Hereditary bleeding disorder: - Congenital hemophilia A without inhibitors with a prophylactic treatment regime - Heterozygous carriers of hemophilia A with subnormal FVIII levels - VWD Type 1, “Vicenza” type Von Willebrand Syndrome (VWS) without specific inhibitors - VWD Type 2b - VWD Type 3 under substitution VWF therapy - Acquired Von Willebrand Syndrome (aVWS) without specific inhibitors 2. Male or female, age = 18 years old at screening 3. If female, must be post-menopausal, surgically sterilized or willing and able to use highly effective methods of birth control throughout the study and for 30 days after the end-of trial (EOT) visit. (NB: Women are the target population for a hemostatic drug because VWD and carrier status of hemophilia causes menorrhagia often severe enough to lead to hysterectomy.) 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 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 25 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects meeting any of the following criteria will be excluded from the study: 1. Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the subject or compromise the quality of the data derived from his/her participation in this study 2. Females who are pregnant (positive pregnancy test at screening or during study phase), lactating, or, if having reproductive potential, are considered potentially unreliable with respect to contraceptive practice 3. History of infusion hypersensitivity reactions, significant drug allergy, or anaphylactic reactions 4. Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the subject to be able to comply fully with study procedures 5. Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the subject’s welfare or the integrity of the study’s results 6. 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: The primary objective of this study is to obtain clinical proof of mechanism for BT200 in one or more hereditary bleeding disorders.;Secondary Objective: The secondary objective of this study is to assess the PK/PD relationship for BT200 in patients with hereditary bleeding disorders. In a pop-PK-sub-study the half-life of the substituted FVIII product with and without BT-200 will be determined. The exploratory objectives of this study are to evaluate the safety, and tolerability of BT200 in patients with hereditary bleeding disorders.;Primary end point(s): Primary endpoint varies according to hematologic diagnosis. - Hemophilia A: increase in FVIII activity - VWD Type 1 & VWS: increase in FVIII activity - VWD Type 2b: increase in platelet count and/or FVIII activity - VWD Type 3 under substitution VWF therapy: increase in trough VWF and FVIII activity levels - aVWS: increase in FVIII activity ;Timepoint(s) of evaluation of this end point: Continuous evaluation for the duration of the subject's participation in the trial | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacokinetics: • Measured concentration of BT200 (and derived PK parameters) • Half-life of the substituted Factor VIII product used with and without BT200 application (pop-PK-sub-study) Pharmacodynamics: • Platelet Function Analyzer (PFA-100®) • Multiplate electrode platelet aggregometer (ristocetin induced) • VWF:ristocetin co-factor assay (VWF:RiCo) • VWF collagen binding assay (VWF:CBA) • Enzyme-linked immunosorbent assay (ELISA) for unbound VWF A1 domain (REAADS®) • VWF propeptide (VWF:pp) • VWF Multimers (non obligatory) • D-dimer and prothrombin fragment (F1.2) • Endogenous thrombin potential (ETP) • Rotational thrombelastometry (ROTEM) • Clot strength assay ;Timepoint(s) of evaluation of this end point: Continuous evaluation for the duration of the subject's participation in the trial | — |
Countries
Austria
Contacts
Public ContactDept. of Clinical Pharmacology
Medical University of Vienna
Outcome results
None listed