Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks

Last updated: August 3, 2015
Sponsor: Pharming Technologies B.V.
Overall Status: Completed

Phase

3

Condition

Hereditary Angioedema

Allergy

Urticaria

Treatment

N/A

Clinical Study ID

NCT01188564
C1 1310
  • Ages > 13
  • All Genders

Study Summary

This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Aged at least 13 years

  • Signed written informed consent

  • Clear clinical and laboratory diagnosis of HAE with baseline plasma level offunctional C1INH of less than 50% of normal

  • Willingness and ability to comply with all protocol procedures

  • Clinical symptoms of an eligible HAE attack with onset less than 5 hours before thetime of initial evaluation

Exclusion

Exclusion Criteria:

  • Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH),or positive anti-rabbit dander IgE test (cut off >0.35 kU/L; ImmunoCap® assay; Phadiaor equivalent).

  • A diagnosis of acquired C1INH deficiency (AAE)

  • Pregnancy, or breastfeeding, or current intention to become pregnant

  • Treatment with any investigational drug in the past 30 days

  • Known or suspected addiction to drug and/or alcohol abuse

  • Suspicion for an alternate explanation of the symptoms other than acute HAE attack

Study Design

Total Participants: 75
Study Start date:
January 01, 2011
Estimated Completion Date:
March 31, 2013

Study Description

HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.

rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.

Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.

Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.

Connect with a study center

  • UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases

    Sofia, 1527
    Bulgaria

    Site Not Available

  • Ottawa Allergy Research Corp.

    Ottawa, Ontario KIY 4G2
    Canada

    Site Not Available

  • Semmelweis University Faculty of Medicine, III Department of Internal Medicine

    Budapest, H-1125
    Hungary

    Site Not Available

  • Allergy, Immunology & Angioedema Center,

    Tel Hashomer, Ramat Gan 52621
    Israel

    Site Not Available

  • Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division

    Haifa, 31048
    Israel

    Site Not Available

  • Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna

    Milan, 20157
    Italy

    Site Not Available

  • P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology

    Skopje, 1000
    Macedonia, The Former Yugoslav Republic of

    Site Not Available

  • Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna

    Krakow, 31-531
    Poland

    Site Not Available

  • Spitalul Clinic JudeÅ£ean MureÅŸ SecÅ£ia Clinică Medicină Internă, Compartimentul de Alergologie ÅŸi Imunologie

    Târgu-Mureş, 540103
    Romania

    Site Not Available

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

    Târgu-Mureş, 540103
    Romania

    Site Not Available

  • Clinic for Immunology and Allergology

    Belgrade, 11000
    Serbia

    Site Not Available

  • Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute

    Mowbray, 7700
    South Africa

    Site Not Available

  • Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre

    Parktown, 2193
    South Africa

    Site Not Available

  • Allergy, Asthma & Immunology, Assoc, Ltd.

    Scottsdale, Arizona 85251
    United States

    Site Not Available

  • Allergy and Asthma Institute of the Valley

    Granada Hills, California 91344
    United States

    Site Not Available

  • UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine

    Los Angeles, California 90095
    United States

    Site Not Available

  • USF Asthma, Allergy and Immunology Clinical Research Unit

    Tampa, Florida 33613
    United States

    Site Not Available

  • Family Allergy and Asthma Center

    Atlanta, Georgia 30342
    United States

    Site Not Available

  • Institute for Asthma and Allergy, P.C.

    Chevy Chase, Maryland 20815
    United States

    Site Not Available

  • Asthma & Allergy Center - Washington University School of Medicine

    St. Louis, Missouri 63141
    United States

    Site Not Available

  • University of Cincinnati Physicians, Inc.

    Cincinnati, Ohio 45267
    United States

    Site Not Available

  • Optimed Research, LTD

    Columbus, Ohio 43235
    United States

    Site Not Available

  • Baker Allergy, Asthma and Dermatology Research Center, LLC

    Lake Oswego, Oregon 97035
    United States

    Site Not Available

  • Pennsylvania State- Milton S. Hershey Medical Center

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • AARA Research Center

    Dallas, Texas 75231
    United States

    Site Not Available

  • University of Texas - Medical Branch

    Galveston, Texas 77555
    United States

    Site Not Available

  • Allergy, Asthma & Immunology Clinic, P.A.

    Irving, Texas 75063
    United States

    Site Not Available

  • Marycliff Allergy Specialists

    Spokane, Washington 99204
    United States

    Site Not Available

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