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

Completed

• Conditions: Hereditary Angioedema
• Interventions: Drug: rhC1INH or pdC1INH

• Registration Number: NCT01397864
• Lead Sponsor: 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 (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.

Detailed Description

see below

Study Timeline

• Study Start: 2011-07
• Study First Submitted: 2011-07-18
• Study First Submitted QC: 2011-07-19
• Study First Posted: 2011-07-20
• Primary Completion: 2024-10-18
• Study Completion: 2024-12-10
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-04
• Last Update Posted: 2025-04-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 181

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Hereditary Angioedema	rhC1INH or pdC1INH	-

Primary Outcome Measures

Name	Time	Method
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	December 2019

Secondary Outcome Measures

Name	Time	Method
To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies)	December 2019

Trial Locations

Locations (17)

UMHAT Alexandrovska EAD; 🇧🇬 Sofia, Bulgaria

KBC Split; 🇭🇷 Split, Croatia

CHC Sestre milosrdnice; 🇭🇷 Zagreb, Croatia

General Hospital Sibenik; 🇭🇷 Šibenik, Croatia

Faculty Hospital by St. Anna Brno; 🇨🇿 Brno, Czech Republic

Hospital A Michallon, CHU Grenoble; 🇫🇷 La Tronche, France

Charite Universitatsmedizin Berlin; 🇩🇪 Berlin, Germany

Semmelweis University, 3rd Department of Internal Medicine; 🇭🇺 Budapest, Hungary

Ospedale Luigi Sacco; 🇮🇹 Milan, Italy

PHI University Clinic of Dermatology; 🇲🇰 Skopje, Macedonia, The Former Yugoslav Republic of

Alesund Hospital; 🇳🇴 Alesund, Norway

Stavanger University Hospital; 🇳🇴 Stavanger, Norway

University Hospital Krakow; 🇵🇱 Krakow, Poland

Bratislava University Hospital; 🇸🇰 Bratislava, Slovakia

University Hospital Martin; 🇸🇰 Martin, Slovakia

University Clinic Golnik; 🇸🇮 Golnik, Slovenia

Ryhof County Hospital; 🇸🇪 Jonkoping, Sweden