Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

This study has been completed.
Information provided by (Responsible Party):
CSL Behring Identifier:
First received: November 7, 2011
Last updated: November 17, 2014
Last verified: November 2014

This is a prospective, international, multi-center, non-randomized, single arm, open-label, postmarketing study to investigate the formation of inhibitory anti-C1-INH antibodies in HAE subjects treated intravenously with Berinert. Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. All subjects will receive 20 U Berinert/kg body weight per attack.

Condition Intervention Phase
Hereditary Angioedema Types I and II
Biological: Berinert, lyophilisate for IV application containing 500 units (U) C1-INH to be reconstituted with 10 mL water for injection
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Prospective Open-label Uncontrolled Multi-center Post-marketing Study to Assess Inhibitory Antibody Formation in Subjects With Congenital C1-INH Deficiency and Acute Hereditary Angioedema (HAE) Attacks Treated With Berinert® , a C1-esterase Inhibitor

Resource links provided by NLM:

Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Incidence of subjects with inhibitory anti-C1-esterase-inhibitor antibodies [ Time Frame: Period of 9 months (baseline to Day 273) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Proportion of subjects with any (inhibitory or non-inhibitory) anti-C1-esterase-inhibitor antibodies [ Time Frame: Period of 9 months (baseline to Day 273) ] [ Designated as safety issue: No ]

Enrollment: 46
Study Start Date: November 2011
Study Completion Date: October 2014
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Berinert Biological: Berinert, lyophilisate for IV application containing 500 units (U) C1-INH to be reconstituted with 10 mL water for injection
Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. Each attack that occurs in this time frame will be treated with 20 U Berinert/kg body weight.


Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of congenital C1-INH deficiency (HAE type I or II) and assessed by the investigator to likely require intravenous (IV) Berinert treatment during the study period.
  • Male or female, ≥ 12 years of age at the time of signing informed consent.
  • Written informed consent for study participation obtained before undergoing any study specific procedures.

Exclusion Criteria:

  • Incurable malignancies in the last 6 months prior to study entry.
  • Acquired angioedema (AAE) due to C1-INH deficiency.
  • All other types of angioedema not associated with C1-INH deficiency.
  • Use of any C1-INH products other than Berinert within 30 days before the study, or planned use during the study.
  • Immunization within 30 days prior to study entry.
  • Autoimmune conditions requiring use of immunosuppressants during the study.
  • Known or suspected hypersensitivity to C1-INH.
  • Participation in any of the previous Berinert studies from which anti-C1-INH antibody results were submitted to the Food and Drug Administration.
  Contacts and Locations
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Please refer to this study by its identifier: NCT01467947

MHAT "Tsaritsa Yoanna"
Sofia, Bulgaria, 1504
Semmelweis University
Budapest, Hungary, 1125
Jagiellonian University
Krakow, Poland, 31-531
Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie
Târgu-Mures, Mureş, Romania, Cod 540103
Sponsors and Collaborators
CSL Behring
Study Director: Mikhail Rojavin CSL Behring
  More Information

No publications provided

Responsible Party: CSL Behring Identifier: NCT01467947     History of Changes
Other Study ID Numbers: CE1145_4001, 2010-024242-30
Study First Received: November 7, 2011
Last Updated: November 17, 2014
Health Authority: United States: Food and Drug Administration
Romania: National Medicines Agency
Bulgaria: Bulgarian Drug Agency
Hungary: National Institute for Quality and Organizational Development in Healthcare and Medicines
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products

Additional relevant MeSH terms:
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Cardiovascular Diseases
Genetic Diseases, Inborn
Hypersensitivity, Immediate
Immune System Diseases
Skin Diseases
Skin Diseases, Vascular
Vascular Diseases processed this record on October 06, 2015