Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

• ClinicalTrials.gov Identifier: NCT01467947
• Recruitment Status: Completed
• First Posted: November 9, 2011
• Results First Posted: November 16, 2015
• Last Update Posted: February 6, 2017
• Sponsor: CSL Behring
• Information provided by (Responsible Party): CSL Behring

Study Description

Brief 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.

Condition or disease	Intervention/treatment	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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 46 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (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
• Study Start Date: November 2011
• Actual Primary Completion Date: October 2014
• Actual Study Completion Date: October 2014

Arms and Interventions

Arm	Intervention/treatment
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.

Outcome Measures

Primary Outcome Measures :

1. Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies [ Time Frame: Baseline to approximately 9 months ]
   Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.

Secondary Outcome Measures :

1. Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies [ Time Frame: Baseline to approximately 9 months ]
   Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies.

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• 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

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

Publications of Results:

Farkas H, Varga L, Moldovan D, Obtulowicz K, Shirov T, Machnig T, Feuersenger H, Edelman J, Williams-Herman D, Rojavin M. Assessment of inhibitory antibodies in patients with hereditary angioedema treated with plasma-derived C1 inhibitor. Ann Allergy Asthma Immunol. 2016 Nov;117(5):508-513. doi: 10.1016/j.anai.2016.08.025. Epub 2016 Oct 24.

• Responsible Party: CSL Behring
• ClinicalTrials.gov Identifier: NCT01467947
• Other Study ID Numbers: CE1145_4001; 2010-024242-30 ( EudraCT Number )
• First Posted: November 9, 2011
• Results First Posted: November 16, 2015
• Last Update Posted: February 6, 2017
• Last Verified: October 2015

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; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Complement C1 Inactivator Proteins; Complement C1 Inhibitor Protein; Complement Inactivating Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs