Clinical trial NCT01397864: C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks
Official title: C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema
Keywords: Hereditary Angioedema
HAE
Angioedema
Recombinant C1 Inhibitor
rhC1INH
registry
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2011-07-20
Enrollment: 181
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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

Eligibility 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)

Primary outcomes:
measure: 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
timeFrame: December 2019
Secondary outcomes:
measure: To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies)
timeFrame: December 2019
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: UMHAT Alexandrovska EAD
city: Split
country: Croatia
facility: KBC Split
city: Zagreb
country: Croatia
facility: CHC Sestre milosrdnice
city: Šibenik
country: Croatia
facility: General Hospital Sibenik
city: Brno
country: Czech Republic
facility: Faculty Hospital by St. Anna Brno
city: La Tronche
country: France
facility: Hospital A Michallon, CHU Grenoble
city: Berlin
country: Germany
facility: Charite Universitatsmedizin Berlin
city: Budapest
country: Hungary
facility: Semmelweis University, 3rd Department of Internal Medicine
city: Milan
country: Italy
facility: Ospedale Luigi Sacco
city: Skopje
country: Macedonia, The Former Yugoslav Republic of
facility: PHI University Clinic of Dermatology
city: Alesund
country: Norway
facility: Alesund Hospital
city: Stavanger
country: Norway
facility: Stavanger University Hospital
city: Krakow
country: Poland
facility: University Hospital Krakow
city: Bratislava
country: Slovakia
facility: Bratislava University Hospital
city: Martin
country: Slovakia
facility: University Hospital Martin
city: Golnik
country: Slovenia
facility: University Clinic Golnik
city: Jonkoping
country: Sweden
facility: Ryhof County Hospital
Study officials:
affiliation: Pharming Technologies BV
name: Anurag Relan, MD
role: STUDY_DIRECTOR
References: