C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01397864
Recruitment Status : Recruiting
First Posted : July 20, 2011
Last Update Posted : April 4, 2018
Information provided by (Responsible Party):
Pharming Technologies B.V.

Brief Summary:
This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.

Condition or disease Intervention/treatment
Hereditary Angioedema Drug: rhC1INH or pdC1INH

Detailed Description:
see below

Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks
Study Start Date : July 2011
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Group/Cohort Intervention/treatment
Hereditary Angioedema Drug: rhC1INH or pdC1INH
C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest)
Other Name: Ruconest

Primary Outcome Measures :
  1. The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INHof acute angioedema attacks [ Time Frame: 3 years ]

Secondary Outcome Measures :
  1. To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies) [ Time Frame: 3 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
The aim is to recruit 300 patients treated with Ruconest. Additionally, the study will continue until 100 patients have been exposed to Ruconest for at least 3 attacks. Enrolment in the pdC1INH arm will be unrestricted.

Inclusion Criteria:

  • Decision to treat the HAE patient with C1 inhibitor (either Ruconest or pdC1INH)
  • Patients must give written informed consent

Exclusion Criteria:

  • A diagnosis of acquired C1INH deficiency (AAE)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01397864

Contact: Tessa Heikamp +31 71 5247 400

UMHAT Alexandrovska EAD Recruiting
Sofia, Bulgaria
Contact: Maria Staevska, Prof.         
Faculty Hospital by St. Anna Brno Recruiting
Brno, Czechia
Contact: Roman Hakl, Dr.         
Hospital A Michallon, CHU Grenoble Recruiting
La Tronche, France
Contact: Laurence Bouillet, Prof. Dr.         
Charite Universitatsmedizin Berlin Recruiting
Berlin, Germany
Contact: Marcus Maurer, Prof.         
Contact: Markus Magerl, Dr.         
Semmelweis University, 3rd Department of Internal Medicine Recruiting
Budapest, Hungary
Contact: Henriette Farkas, Prof.         
Ospedale Luigi Sacco Recruiting
Milan, Italy
Contact: Marco Cicardi, Prof.         
Macedonia, The Former Yugoslav Republic of
PHI University Clinic of Dermatology Recruiting
Skopje, Macedonia, The Former Yugoslav Republic of
Contact: Vesna Grivcheva-Panovska, Prof.         
Alesund Hospital Recruiting
Alesund, Norway
Contact: Robert Brudevold, Dr.         
Stavanger University Hospital Recruiting
Stavanger, Norway
Contact: Ellen Pritzier, Dr.         
University Hospital Krakow Recruiting
Krakow, Poland
Contact: Krystyna Obtulowicz, Prof.         
Bratislava University Hospital Recruiting
Bratislava, Slovakia
Contact: Katarina Hrubiskova, Dr.         
University Hospital Martin Recruiting
Martin, Slovakia
Contact: Milos Jesenak, Prof.         
Ryhof County Hospital Recruiting
Jonkoping, Sweden
Contact: Janne Bjorkander, Prof.         
Sponsors and Collaborators
Pharming Technologies B.V.
Study Director: Anurag Relan, MD Pharming Technologies BV

Responsible Party: Pharming Technologies B.V. Identifier: NCT01397864     History of Changes
Other Study ID Numbers: C1 1412
First Posted: July 20, 2011    Key Record Dates
Last Update Posted: April 4, 2018
Last Verified: April 2018

Keywords provided by Pharming Technologies B.V.:
Hereditary Angioedema
Recombinant C1 Inhibitor

Additional relevant MeSH terms:
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Immune System Diseases
Genetic Diseases, Inborn
Complement C1 Inactivator Proteins
Complement C1 Inhibitor Protein
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs