Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

NCT01467947 | Completed Phase 4 Results

• 2 other identifiers: CE1145_40012010-024242-30
• Study Type: interventional
• Target: 46
• Locations: 4 countries
• Sites: 4

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.

Conditions

Hereditary Angioedema Types I and II

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to approximately 9 months

Secondary Outcomes (1)

• Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies

  Baseline to approximately 9 months

Study Arms (1)

Berinert

EXPERIMENTAL

Biological: Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection

Interventions

Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection BIOLOGICAL

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.

Berinert

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (4)

MHAT "Tsaritsa Yoanna"

Sofia, 1504, Bulgaria

Location

Semmelweis University

Budapest, 1125, Hungary

Location

Jagiellonian University

Krakow, Krakow, 31-531, Poland

Location

Spitalul Clinic Judeţean Mureş,Secţia Clinică Medicină Internă,Compartimentul Alergologie şi Imunologie

Târgu-Mures, Mureș County, Cod 540103, Romania

Location

Related Publications (1)

• 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.

  PMID: 27788880 RESULT

MeSH Terms

Conditions

Hereditary Angioedema Types I and II

Interventions

Complement C1 Inhibitor Protein; Water; Injections

Condition Hierarchy (Ancestors)

Angioedemas, Hereditary; Angioedema; Vascular Diseases; Cardiovascular Diseases; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Complement C1 Inactivator Proteins; Serpins; Peptides; Amino Acids, Peptides, and Proteins; Complement Inactivator Proteins; Complement System Proteins; Immunoproteins; Blood Proteins; Proteins; Hydroxides; Alkalies; Inorganic Chemicals; Anions; Ions; Electrolytes; Oxides; Oxygen Compounds; Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Program Director, Clinical R&D
• Organization: CSL Behring

clinicaltrials@cslbehring.com

Use email contact

Study Officials

• Mikhail Rojavin

  CSL Behring

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2011
• First Posted: November 9, 2011
• Study Start: November 1, 2011
• Primary Completion: October 1, 2014
• Study Completion: October 1, 2014
• Last Updated: February 6, 2017
• Results First Posted: November 16, 2015

Record last verified: 2015-10

Locations

BU (1); HU (1); RO (1); PL (1)