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

April 4, 2025 updated by: Pharming Technologies B.V.

C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks

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 (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

see below

Study Type

Observational

Enrollment (Actual)

181

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Bulgaria
      • Sofia, Bulgaria
        • UMHAT Alexandrovska EAD
  • Croatia
      • Split, Croatia
        • KBC Split
      • Zagreb, Croatia
        • CHC Sestre milosrdnice
      • Šibenik, Croatia
        • General Hospital Sibenik
  • Czech Republic
      • Brno, Czech Republic
        • Faculty Hospital by St. Anna Brno
  • France
      • La Tronche, France
        • Hospital A Michallon, CHU Grenoble
  • Germany
      • Berlin, Germany
        • Charité Universitätsmedizin Berlin
  • Hungary
      • Budapest, Hungary
        • Semmelweis University, 3rd Department of Internal Medicine
  • Italy
      • Milan, Italy
        • Ospedale Luigi Sacco
  • Macedonia, The Former Yugoslav Republic of
      • Skopje, Macedonia, The Former Yugoslav Republic of
        • PHI University Clinic of Dermatology
  • Norway
      • Alesund, Norway
        • Ålesund Hospital
      • Stavanger, Norway
        • Stavanger University Hospital
  • Poland
      • Krakow, Poland
        • University Hospital Krakow
  • Slovakia
      • Bratislava, Slovakia
        • Bratislava University Hospital
      • Martin, Slovakia
        • University Hospital Martin
  • Slovenia
      • Golnik, Slovenia
        • University Clinic Golnik
  • Sweden
      • Jonkoping, Sweden
        • Ryhof County Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

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.

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INH of acute angioedema attacks
Time Frame: December 2019
December 2019

Secondary Outcome Measures

Outcome Measure
Time Frame
To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies)
Time Frame: December 2019
December 2019

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pharming Technologies B.V.

Investigators

  • Study Director: Anurag Relan, MD, Pharming Technologies BV

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2011

Primary Completion (Actual)

October 18, 2024

Study Completion (Actual)

December 10, 2024

Study Registration Dates

First Submitted

July 18, 2011

First Submitted That Met QC Criteria

July 19, 2011

First Posted (Estimated)

July 20, 2011

Study Record Updates

Last Update Posted (Actual)

April 8, 2025

Last Update Submitted That Met QC Criteria

April 4, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • C1 1412

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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