Clinical trial NCT01467947: Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hereditary Angioedema Types I and II
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2011-11-09
Enrollment: 46
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies
timeFrame: Baseline to approximately 9 months
Secondary outcomes:
measure: Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies
timeFrame: Baseline to approximately 9 months
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: MHAT "Tsaritsa Yoanna"
city: Budapest
country: Hungary
facility: Semmelweis University
city: Krakow
country: Poland
facility: Jagiellonian University
city: Târgu-Mures
country: Romania
facility: Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie
state: Mureş
Study officials:
affiliation: CSL Behring
name: Mikhail Rojavin
role: STUDY_DIRECTOR
References:
citation: 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.