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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2015 by Pharming Technologies B.V.
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.
ClinicalTrials.gov Identifier:
NCT01397864
First received: July 18, 2011
Last updated: February 15, 2016
Last verified: November 2015
  Purpose
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 Intervention
Hereditary Angioedema
Drug: rhC1INH or pdC1INH

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Pharming Technologies B.V.:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies) [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 300
Study Start Date: July 2011
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Hereditary Angioedema Drug: rhC1INH or pdC1INH
C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest)
Other Name: Ruconest

Detailed Description:
see below
  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
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.
Criteria

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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01397864

Contacts
Contact: Tessa Heikamp +31 71 5247 400 t.heikamp@pharming.com

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

Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT01397864     History of Changes
Other Study ID Numbers: C1 1412 
Study First Received: July 18, 2011
Last Updated: February 15, 2016
Health Authority: European Union: European Medicines Agency

Keywords provided by Pharming Technologies B.V.:
Hereditary Angioedema
HAE
Angioedema
Recombinant C1 Inhibitor
rhC1INH
registry

Additional relevant MeSH terms:
Angioedemas, Hereditary
Angioedema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
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

ClinicalTrials.gov processed this record on August 24, 2016