Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Hereditary Angioedema Types I and II Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01467947
• Other study ID #: CE1145_4001
• Secondary ID: 2010-024242-30
• Status: Completed
• Phase: Phase 4
• First received: November 7, 2011
• Last updated: October 16, 2015
• Start date: November 2011
• Est. completion date: October 2014

Study information

• Verified date: October 2015
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationRomania: National Medicines AgencyBulgaria: Bulgarian Drug AgencyHungary: National Institute for Quality and Organizational Development in Healthcare and MedicinesPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 46
• Est. completion date: October 2014
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema Types I and II

Intervention

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.

Locations

Country	Name	City	State
Bulgaria	MHAT "Tsaritsa Yoanna"	Sofia
Hungary	Semmelweis University	Budapest
Poland	Jagiellonian University	Krakow
Romania	Spitalul Clinic Judetean Mures,Sectia Clinica Medicina Interna,Compartimentul Alergologie si Imunologie	Târgu-Mures	Mures

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Bulgaria,  Hungary,  Poland,  Romania, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies	Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.	Baseline to approximately 9 months	No
Secondary	Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies	Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies.	Baseline to approximately 9 months	No