Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert
Primary Purpose
Hereditary Angioedema Types I and II
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Angioedema Types I and II
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.
Sites / Locations
- MHAT "Tsaritsa Yoanna"
- Semmelweis University
- Jagiellonian University
- Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Berinert
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01467947
Brief Title
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
2. Study Status
Record Verification Date
October 2015
Overall Recruitment Status
Completed
Study Start Date
November 2011 (undefined)
Primary Completion Date
October 2014 (Actual)
Study Completion Date
October 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring
4. Oversight
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema Types I and II
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
46 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Berinert
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies
Description
Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.
Time Frame
Baseline to approximately 9 months
Secondary Outcome Measure Information:
Title
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies
Description
Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies.
Time Frame
Baseline to approximately 9 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mikhail Rojavin
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
MHAT "Tsaritsa Yoanna"
City
Sofia
ZIP/Postal Code
1504
Country
Bulgaria
Facility Name
Semmelweis University
City
Budapest
ZIP/Postal Code
1125
Country
Hungary
Facility Name
Jagiellonian University
City
Krakow
ZIP/Postal Code
31-531
Country
Poland
Facility Name
Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie
City
Târgu-Mures
State/Province
Mureş
ZIP/Postal Code
Cod 540103
Country
Romania
12. IPD Sharing Statement
Citations:
PubMed Identifier
27788880
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.
Results Reference
result
Learn more about this trial
Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert
We'll reach out to this number within 24 hrs