Clinical trial NCT02854397: Determination of Specific Biomarkers of Acute Attack of Angioedema Within Pediatric Population
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hereditary Angioedema
Healthy Volunteers
Lead sponsor: University Hospital, Grenoble
Sponsor class: OTHER
First posted: 2016-08-03
Enrollment: 31
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: patients with hereditary angioedema patients with angioedema resulting of mast cell activation healthy patients, without angioedema

Brief summary: In emergency room, this is crucial to diagnose an acute attack of hereditary angioedema (HAE) to quickly provide the efficient treatment. Currently, there is no specific biomarker for acute attack of bradykinin-mediated angioedema to help clinicians for patient care. However, previous works are carried out for that purpose. All the potential candidate biomarkers must be validated in prospective studies to estimate their specificity and sensitivity values, and to understand their potential utility in patient care.

The main goal of this clinical trial is to estimate the diagnostic value of VE-cadherin in pediatric population, for the differential diagnosis between HAE crisis and angioedema resulting of mast cell activation crisis (the main differential diagnosis of HAE).


Eligibility criteria: Inclusion Criteria:

For HAE: patient with a documented diagnosis of HAE:

* type I (from an antigenic deficiency of the C1 esterase inhibitor) or type II (from a functional deficiency of the C1 esterase inhibitor). The existence of a mutation in SERPING1 was not necessary for the inclusion
* HAE with normal C1-INH (ex type III) with a required mutation in FXII gene or with a typical family history of HAE diagnosed by a specialized physician belonging to CREAK network.

For AE resulting of mast cell activation: a documented diagnosis of AE resulting of mast cell activation included:

* mastocytosis,
* chronic spontaneous urticaria,
* acute urticaria after exposure of allergen during allergy challenge tests,
* mast cell activation syndrome.

For the control group:

* composed of patients who presented a stabilized disease (that was not infectious, not auto-inflammatory or inflammatory disease and without implication of endothelial cells).

Exclusion Criteria:

* Over 18 years or under 1 year.
* Diagnosis of HAE with a normal C1 esterase inhibitor or AE of unknown aetiology.
* Patients with HAE who received an acute attack treatment before the blood sample (the C1 esterase inhibitor concentrate or a bradykinin B2 receptor antagonist); patients with HAE who received a prophylactic treatment (danazol).
* Patients who were treated by omalizumab or corticosteroid treatment.

Primary outcomes:
measure: VE-cadherin level
timeFrame: Half a day
Secondary outcomes:
measure: Dosage of VE-cadherin (vascular endothelial)
timeFrame: Half a day
measure: Dosage of Fc KHPM
timeFrame: Half a day
measure: Dosage of D-dimer
timeFrame: Half a day
measure: Dosage of Tryptase
timeFrame: Half a day
Other outcomes:
Locations:
city: Angers
country: France
facility: University hospital angers
city: Besançon
country: France
facility: University Hospital Besançon
city: Bordeaux
country: France
facility: University hospital Bordeaux
city: Clermont-Ferrand
country: France
facility: University hopital Clermont-Ferrand
city: Grenoble
country: France
facility: University Hospital Grenoble
city: Lille
country: France
facility: University Hospital Lille
city: Lyon
country: France
facility: University Hospital Lyon
city: Marseille
country: France
facility: University hospital Marseille
city: Montpellier
country: France
facility: University hospital Montpellier
city: Nancy
country: France
facility: University hospital Nancy
city: Niort
country: France
facility: General Hospital
city: Paris
country: France
facility: university hospital Saint-Antoine (AP-HP)
city: Rouen
country: France
facility: University hospital Rouen
city: Toulouse
country: France
facility: University hospital Toulouse
Study officials:
affiliation: University Hospital, Grenoble
name: Anne Pagnier
role: PRINCIPAL_INVESTIGATOR
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