Patient Registry Study of Berinert® in Normal Clinical Practice

Patient Registry for Berinert®, a C1-Esterase Inhibitor

The objective of this patient registry is to collect data on the safety of Berinert® in normal clinical practice in the United States. The patient registry will be maintained for a period of at least 3 years. The duration of individual patient participation will vary and is determined by the frequency of hereditary angioedema (HAE) attacks and the patient's need for Berinert® treatment.

Study Overview

• Status: Completed
• Conditions: Includes: Hereditary Angioedema
• Intervention / Treatment: Biological: Berinert® (C1 Esterase Inhibitor)

• Study Type: Observational
• Enrollment (Actual): 318

Contacts and Locations

Study Locations

• Denmark
  • Odense C, Denmark, 5000
    • Denmark, Odense C
• Germany
  • Berlin, Germany, 10117
    • Germany, Berlin
  • Frankfurt, Germany, 60596
    • Germany, Frankfurt
  • Mainz, Germany, 55101
    • Germany, Mainz
  • München, Germany, 81675
    • Germany, München
• Switzerland
  • Luzern 16, Switzerland, CH-6000
    • Switzerland, Luzern 16
• United States
  • Arizona
    • Scottsdale, Arizona, United States, 85251
      • United States, Arizona
  • California
    • Granada Hills, California, United States, 91344
      • United States, California
    • Los Angeles, California, United States, 90095
      • United States, California
    • Redding, California, United States, 96003
      • United States, California
    • San Diego, California, United States, 92093
      • United States, California
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • United States, Georgia
  • Idaho
    • Idaho Falls, Idaho, United States, 83404
      • United States, Idaho
  • Louisiana
    • New Orleans, Louisiana, United States, 70118
      • United States, Louisiana
  • Maryland
    • Chevy Chase, Maryland, United States, 20815
      • United States, Maryland
    • Columbia, Maryland, United States, 21044
      • United States, Maryland
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • United States, Massachusetts
  • Minnesota
    • Plymouth, Minnesota, United States, 55446
      • United States, Minnesota
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • United States, Missouri
    • St. Louis, Missouri, United States, 63110
      • United States, Missouri
  • New Jersey
    • Branchburg, New Jersey, United States, 08876
      • United States, New Jersey
    • Edison, New Jersey, United States, 88220
      • United States, New Jersey
    • Iselin, New Jersey, United States, 08830
      • United States, New Jersey
    • Ocean, New Jersey, United States, 07712
      • United States, New Jersey
  • New York
    • New York, New York, United States, 10029
      • United States, New York
    • North Syracuse, New York, United States, 13212
      • United States, New York
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • United States, North Carolina
  • Ohio
    • Centerville, Ohio, United States, 45458
      • United States, Ohio
    • Cincinnati, Ohio, United States, 45267
      • United States, Ohio
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73131
      • United States, Oklahoma
    • Tulsa, Oklahoma, United States, 74133
      • United States, Oklahoma
  • Oregon
    • Lake Oswego, Oregon, United States, 97035
      • United States, Oregon
  • Pennsylvania
    • Altoona, Pennsylvania, United States, 16601
      • United States, Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • United States, Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • United States, Pennsylvania
  • South Carolina
    • Columbia, South Carolina, United States, 29203
      • United States, South Carolina
  • South Dakota
    • Rapid City, South Dakota, United States, 57702
      • United States, South Dakota
  • Texas
    • Dallas, Texas, United States, 75231
      • United States, Texas
  • Washington
    • Spokane, Washington, United States, 99204
      • United States, Washington
    • Tacoma, Washington, United States, 98405
      • United States, Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

An attempt will be made to prospectively identify patients and enroll them into this registry before treatment is required. In addition, retrospective chart/case review and data collection may be conducted.

Description

Inclusion Criteria:

• Any patient receiving CSL Behring's C1-esterase inhibitor
• Written informed consent (may not be required for some retrospective chart review cases)

Exclusion Criteria:

• Any patient participating in an HAE study using other C1-inhibitors than Berinert®

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Berinert; Patients requiring treatment with Berinert®	Biological: Berinert® (C1 Esterase Inhibitor); Berinert® is marketed as a lyophilized concentrate available in a single-use vial to be reconstituted with sterile water before administration as described in the prescribing information.; Other Names: Berinert P; CE1145; C1-INH

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of thrombotic and thrombo-embolic events	Within 30 days of treatment with Berinert®
Occurrence of suspected viral transmission	Duration of the study, at least 3 years
Use of concomitant medications and plasma products	Duration of the study, at least 3 years

Collaborators and Investigators

• Sponsor: CSL Behring
• Collaborators: Chiltern International Inc.

Publications and helpful links

General Publications

• Bygum A, Martinez-Saguer I, Bas M, Rosch J, Edelman J, Rojavin M, Williams-Herman D; Berinert Registry Investigators. Use of a C1 Inhibitor Concentrate in Adults >/=65 Years of Age with Hereditary Angioedema: Findings from the International Berinert(R) (C1-INH) Registry. Drugs Aging. 2016 Nov;33(11):819-827. doi: 10.1007/s40266-016-0403-0.
• Busse P, Bygum A, Edelman J, Lumry W, Machnig T, Martinez-Saguer I, Rojavin M. Safety of C1-esterase inhibitor in acute and prophylactic therapy of hereditary angioedema: findings from the ongoing international Berinert patient registry. J Allergy Clin Immunol Pract. 2015 Mar-Apr;3(2):213-9. doi: 10.1016/j.jaip.2014.08.014. Epub 2014 Oct 29.

Helpful Links

• Hereditary Angioedema Association (HAEA)

Study record dates

Study Major Dates

• Study Start: April 1, 2010
• Primary Completion (ACTUAL): April 1, 2014
• Study Completion (ACTUAL): April 1, 2014

Study Registration Dates

• First Submitted: April 12, 2010
• First Submitted That Met QC Criteria: April 21, 2010
• First Posted (ESTIMATE): April 22, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): June 9, 2014
• Last Update Submitted That Met QC Criteria: June 5, 2014
• Last Verified: June 1, 2014

More Information

Terms related to this study

• Keywords: Hereditary angioedema; Acute HAE attack; C1-esterase Inhibitor
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary; Physiological Effects of Drugs; Immunosuppressive Agents; Immunologic Factors; Complement Inactivating Agents; Complement C1 Inhibitor Protein; Complement C1 Inactivator Proteins; Complement C1s
• Other Study ID Numbers: CE1145_5002; 1500 (CSL Behring)