Patients with hereditary angioedema (HAE) suffer from recurring and mostly unforeseeable attacks of acute oedema of subcutaneous tisses of various organs. The pathophysiological correlate of this disease is a deficiency in functionally active C1-Esterase Inhibitor (C1-INH). The defects in active C1-Esterase Inhibitor are inherited as an autosomal dominant trait.
Conditions
Interventions
Trade Name: Berinert P
Pharmaceutical Form: Concentrate and solvent for solution for injection
INN or Proposed INN: C1-Esterase-Inhibitor
Concentration unit: U unit(s)
Concentration type: equal
Conce
Sponsors
Fachbereich Medizin der Johann Wolfgang Goethe-Universität Frankfurt/M.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects with an established diagnosis of Hereditary Angioedema (HAE)type I (C1-Inhibitor activity =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects without an established diagnosis of HAE Last C1-INH administration less than 7 days ago and/or acute attack Subjects with acquired angioedeme (AAE) All other types of angioedema not associated with C1-INH deficiency Treatment with any investigational drug (exclusive drugs appropriate for the treatment of acute angioedema) 30 days before study treatment Treatment with any other drug appropriate for the treatment of acute angioedema within 2 weeks before start of study treatment at each phase Danazol prophylaxis Prophylaxis with antifibrinolytics, EACA, tranexamic acid Subjects with a known hypersensitivity to study medication (Berinert P) Pregnant women (pregnancy rapid assay required for women with childbearing potential), women currently breast-feeding, or with the intention to breast-feed Subjects with malignant diseases Subjects with immunodeficiencies such as established acquired immunodeficiency syndrome Subjects with concurrent serious or acute illness or infection as per investigators judgement. Subjects with mental conditions which render the subject or its legally acceptable representative unable to understand the nature, scope and possible consequences of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate and compare pharmacokinetics of study medication (Berinert P) in subjects with hereditary angioedema after subcutaneous and intravenous administration;Secondary Objective: Documentation of adverse events Screening for C1-Inhibitor antibodies Investigation of virus markers ;Primary end point(s): To investigate and compare pharmacokinetics of study medication (Berinert P) in HAE subjects after subcutaneous and intravenous administration Individual courses of C1-inhibitor levels, from these will be derived: Area under the curve (AUC; for dose of 1,000 U per subject [(U x hour)/mL] Time to maximum concentration (Tmax; hours) Maximum concentration (Cmax) Terminal elimination half-life (t1/2) Mean residence time (MRT; hours) Total clearance (Cl; mL/[kg x hour]) Volume of distribution at steady state (Vss; mL/kg) In-vivo recovery (IVR) Classical IVR (% rise/U/ml) Incremental IVR (response) (% rise/U/kg body weight) | — |
Countries
Germany
Outcome results
None listed