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Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01467947
First received: November 7, 2011
Last updated: October 16, 2015
Last verified: October 2015
  Purpose
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 IU Berinert/kg body weight per attack.

Condition Intervention Phase
Hereditary Angioedema Types I and II
Biological: Berinert, lyophilizate for IV application containing 500 IU 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:
  • Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies [ Time Frame: Baseline to approximately 9 months ] [ Designated as safety issue: No ]
    Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.


Secondary Outcome Measures:
  • Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies [ Time Frame: Baseline to approximately 9 months ] [ Designated as safety issue: No ]
    Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies.


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, lyophilizate for IV application containing 500 IU 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 IU Berinert/kg body weight.

  Eligibility

Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01467947

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

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01467947     History of Changes
Other Study ID Numbers: CE1145_4001  2010-024242-30 
Study First Received: November 7, 2011
Results First Received: October 16, 2015
Last Updated: October 16, 2015
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:
Angioedema
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement C1 Inactivator Proteins
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 26, 2016