Back to results

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

About this trial

This is an interventional treatment trial for Hereditary Angioedema Types I and II

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

NCT ID

NCT01467947

First Posted

November 7, 2011

Last Updated

December 12, 2016

Sponsor

CSL Behring

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

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