PHARMACOKINETIC BERINERT P STUDY OF SUBCUTANEOUS VERSUS INTRAVENOUS ADMINISTRATION IN SUBJECTS WITH MODERATE HEREDITARY ANGIOEDEMA - THE PASSION STUDY - PASSION STUDY

Conditions: 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.

Registration Number: EUCTR2008-000654-12-DE

Lead Sponsor: Fachbereich Medizin der Johann Wolfgang Goethe-Universität Frankfurt/M.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

Subjects with an established diagnosis of Hereditary Angioedema (HAE)type I (C1-Inhibitor activity <50% and C1-Inhibitor antigen < 15.4 mg/dl) or HAE type II (C1-Inhibitor activity < 50% and C1-Inhibitor antigen in normal or elevated concentration of dysfunctional protein).
Male and female subjects with an age of 18 years
Subjects providing an informed consent

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

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>Screening for C1-Inhibitor antibodies<br>Investigation of virus markers <br>;Primary end point(s): To investigate and compare pharmacokinetics of study medication (Berinert P) in HAE subjects after subcutaneous and intravenous administration<br>Individual courses of C1-inhibitor levels, from these will be derived: <br><br>Area under the curve (AUC; for dose of 1,000 U per subject [(U x hour)/mL]<br>Time to maximum concentration (Tmax; hours)<br>Maximum concentration (Cmax)<br>Terminal elimination half-life (t1/2)<br>Mean residence time (MRT; hours)<br>Total clearance (Cl; mL/[kg x hour])<br>Volume of distribution at steady state (Vss; mL/kg)<br>In-vivo recovery (IVR) <br>Classical IVR (% rise/U/ml)<br>Incremental IVR (response) (% rise/U/kg body weight)<br>

Secondary Outcome Measures