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CompletedNCT01397864Updated Apr 8, 2025

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

An observational study in Hereditary Angioedema, sponsored by Pharming Technologies B.V.. Completed at 17 sites in 13 countries. Per ClinicalTrials.gov, last updated 2025-04-08.

Sponsored by Pharming Technologies B.V. · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
181
Sex
All
01

Study 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.

Read the detailed description

see below

02

Conditions studied

  • Hereditary Angioedema

Keywords

  • Hereditary Angioedema
  • HAE
  • Angioedema
  • Recombinant C1 Inhibitor
  • rhC1INH
  • registry
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
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

Exclusion Criteria:

  • A diagnosis of acquired C1INH deficiency (AAE)
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
181 participants (actual)
Patient registry
No

Groups and cohorts

  • Hereditary Angioedema

    Drug: rhC1INH or pdC1INH

Interventions

  • DrugrhC1INH or pdC1INH

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

    Also known as: Ruconest

05

What researchers measure

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

    Time frame: December 2019

Secondary outcomes

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

    Time frame: December 2019

06

Study locations

17 sites
  • 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
07

Registry details

Key details

Study ID
NCT01397864
Lead sponsor
Pharming Technologies B.V.
Responsible party
Sponsor
First posted
Jul 20, 2011
Start date
Jul 2011
Primary completion
Oct 18, 2024
Completion
Dec 10, 2024
Last update
Apr 8, 2025

Study contacts

Anurag Relan, MD
study director · Pharming Technologies BV

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.

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