Pharmacokinetics and Safety of Human Pasteurised C1-Inhibitor Concentrate (Berinert/CE1145) in Subjects with Congenital C1-INH Deficiency.

Conditions: Hereditary Angioedema (HAE)
MedDRA version: 14.1Level: LLTClassification code 10056912Term: C1 esterase inhibitor deficiencySystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2010-019670-32-IT

Lead Sponsor: AZIENDA OSPEDALIERA L. SACCO (A.O. DI RILIEVO NAZIONALE)

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

Patients who suffer from frequent angioedema attacks (every 3-7 days) in whom infused CE1145 is rapidly catabolized and does not significantly modify C4 levels. Written informed consent for study participation obtained before undergoing any study specific procedures.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

•Absence of HAE attack in the last 30 days before study enrolment.
•Participation in another clinical study (or use of another investigational medicinal product [IMP]) within 30 days before, or during, the study.
•History of alcohol, drug, or medication abuse within one year before the study.
•Use of concomitant therapy not permitted during the study.
•Suspected inability (eg, language problems) or unwillingness to comply with study procedures.
•Mental condition rendering the subject (or the subject’s legally acceptable representative[s]) unable to understand the nature, scope and possible consequences of the study).
•Known or suspected hypersensitivity to the IMP, or to any excipients of the IMP.
•Known or suspected antibodies to the IMP, or to any excipients of the IMP.
•Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct 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 the PK profile of CE1145 in patients with weekly angioedema attacks and rapid clearance of C1-INH from plasma after normalising activation of the complement and contact system by replacing the total C1-esterase inhibitor (C1-INH) plasma pool with repetitive C1-INH administrations.;Secondary Objective: The secondary objective of the study is to assess the safety of C1-INH treatment in these subjects.;Primary end point(s): PK of i.v. administration of study medication at steady state under normalised activity of C1-INH.;Timepoint(s) of evaluation of this end point: Week 9 of the study.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Safety of i.v. administration of study medication at high doses.;Timepoint(s) of evaluation of this end point: Week 9 of the study.