NCT01397864

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.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
181

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jul 2011

Longer than P75 for all trials

Geographic Reach
11 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2011

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

July 18, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 20, 2011

Completed
13.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 18, 2024

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 10, 2024

Completed
Last Updated

April 8, 2025

Status Verified

April 1, 2025

Enrollment Period

13.3 years

First QC Date

July 18, 2011

Last Update Submit

April 4, 2025

Conditions

Keywords

Hereditary AngioedemaHAEAngioedemaRecombinant C1 InhibitorrhC1INHregistry

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies)

    December 2019

Study Arms (1)

Hereditary Angioedema

Drug: rhC1INH or pdC1INH

Interventions

C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest)

Also known as: Ruconest
Hereditary Angioedema

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability 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.

You may qualify if:

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

You may not qualify if:

  • A diagnosis of acquired C1INH deficiency (AAE)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

UMHAT Alexandrovska EAD

Sofia, Bulgaria

Location

KBC Split

Split, Croatia

Location

General Hospital Sibenik

Šibenik, Croatia

Location

CHC Sestre milosrdnice

Zagreb, Croatia

Location

Faculty Hospital by St. Anna Brno

Brno, Czechia

Location

Hospital A Michallon, CHU Grenoble

La Tronche, France

Location

Charite Universitatsmedizin Berlin

Berlin, Germany

Location

Semmelweis University, 3rd Department of Internal Medicine

Budapest, Hungary

Location

Ospedale Luigi Sacco

Milan, Italy

Location

PHI University Clinic of Dermatology

Skopje, North Macedonia

Location

Alesund Hospital

Ålesund, Norway

Location

Stavanger University Hospital

Stavanger, Norway

Location

University Hospital Krakow

Krakow, Poland

Location

Bratislava University Hospital

Bratislava, Slovakia

Location

University Hospital Martin

Martin, Slovakia

Location

University Clinic Golnik

Golnik, Slovenia

Location

Ryhof County Hospital

Jönköping, Sweden

Location

MeSH Terms

Conditions

Angioedemas, HereditaryAngioedema

Interventions

conestat alfa

Condition Hierarchy (Ancestors)

Vascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency Syndromes

Study Officials

  • Anurag Relan, MD

    Pharming Technologies BV

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 18, 2011

First Posted

July 20, 2011

Study Start

July 1, 2011

Primary Completion

October 18, 2024

Study Completion

December 10, 2024

Last Updated

April 8, 2025

Record last verified: 2025-04

Locations