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Identification and Characterization of Genetic Variants in Hereditary Angioedema

Not yet recruiting
Conditions
Hereditary Angioedema With C1 Esterase Inhibitor Deficiency
Registration Number
NCT05833620
Lead Sponsor
Hospital Universitari Vall d'Hebron Research Institute
Brief Summary

This project aims to analyse in an unbiased way the existence of genetic variants that contribute to explaining and predicting the differences in clinical expression between patients with HAE.

Detailed Description

Not available

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
200
Inclusion Criteria
  • Adult patients (≥ 18 years old) with HAE-C1INH diagnosis (confirmed by mutation in SERPING1 gen or immunochemical study showing a decrease in C1INH function <50% in two determinations together with a family history (symptomatic patients' group)
  • Patients ≥ 22 years old with C1INH hereditary deficiency (confirmed by mutation of SERPING1 gene or immunochemical study showing a decrease in C1INH function <50% in two determinations together with a family history) and who have not developed symptoms consistent with HAE-C1INH
  • Signed informed consent.
Exclusion Criteria
  • No confirmed C1INH deficiency.
  • Inability to sign the informed consent.
  • Presence of recurrent angioedema with histaminergic characteristics (response to treatment with antihistamines, glucocorticoids and/or epinephrine)

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Set of validated disease-modifying genetic variants in Spanish patients with HAE-C1INHDay 1

To identify and characterize novel genetic variants associated with the incomplete penetrance and variable clinical expressivity observed in HAE-C1INH patients.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (2)

Hospital Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital Universitario La Paz

🇪🇸

Madrid, Spain

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