Human pasteurized C1 esterase inhibitor concentrate (CE1145) in subjects with congenital C1-INH deficiency and acute abdominal or facial HAE attacks - C1-INH HAE study

Phase 1

• Conditions: Hereditary angioedema (HAE) is characterized by congenital C1-INH deficiency. This is reflected by insufficient plasma concentrations of C1 – inhibitor or by synthesis of dysfunctional C1 – inhibitor molecules, which can lead to attacks of abdominal pain and/or severe swelling in the tissues e.g. of the face. Replacement with C1 esterase inhibitor concentrate is the treatment of choice of an acute attack of HAE.; MedDRA version: 9.1Level: LLTClassification code 10019860Term: Hereditary angioedema; MedDRA version: 9.1Level: PTClassification code 10019860Term: Hereditary angioedema

• Registration Number: EUCTR2004-001186-17-SE
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2006-02-02
• Study Completion: 2007-12-28
• Last Update Posted: 24 January 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 126

Inclusion Criteria

- Male and female subjects >= 6 years
- Documented congenital C1-INH deficiency with immunogenic and/or functional C1-INH level below the lower limit of the laboratory reference range (HAE type I or II); to be confirmed by central laboratory.
- Documented history of abdominal or facial attacks
- Acute abdominal or facial attack with moderate /severe intensity for no longer than
5.0 hours at the time of study medication administration
- Informed written consent has been obtained (signed by subject, or, in case of a minor, by his/her legally acceptable representative(s))

Are the trial subjects under 18? yes
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

- Life expectancy < 6 months
- Incurable malignancies with metastases
- History of hypersensitivity to the study medication
- Acquired angioedema due to C1-INH deficiency (e. g., onset at age > 40 years, no family history of congenital C1-INH deficiency, low C1q level, and no known HAE mutation)
- All other types of angioedema not associated with C1-INH deficiency
(including e.g. anaphylactic angioedema)
- Abdominal pain caused by other pathology (e. g., appendicitis, myocardial infarction)
- End-stage liver disease (i.e., Child-Pugh-score B or C)
- Fever (> 38.3°C or 101.0 F)
- High white blood cell count (> 20.0 x 10 exp9/L )
- Pregnant women (pregnancy test required), women currently breast-feeding, or with the intention to breast feed
- Treatment with any other investigational drug (except IMP and any other drugs appropriate for the treatment of acute angioedema) 30 days before start of study treatment on Day 1.
- Treatment with any C1-INH concentrate or any other drugs appropriate for the treatment of acute angioedema within 7 days before start of study treatment on Day 1.
- Treatment with fresh frozen plasma or native plasma within 7 days before start of study treatment on Day 1.
- Treatment with ACE inhibitors within 4 weeks before start of study treatment on Day 1.
- Narcotic pain medication and/or anti-emetics between start of attack and administration of study medication.
- Evidence of narcotic seeking behavior and/or drug addiction (including alcohol abuse)
- 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
- Prior inclusion into this study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified