The objective of the study is to estimate the utility and the minimally clinically significant symptom difference in VAS and verbal descriptor scores as the basis for the onset of relief for skin swelling, skin pain or abdominal pain in patients…
ID
Source
Brief title
Condition
- Immune system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Descriptive research based on which the "minimally clinically significant
difference" in symptomscore can be assessed.
Secondary outcome
not applicable
Background summary
Hereditary angio-edema is a congenital disease due to a deficiency of
C1-esterase inhibitor. It may lead to angio-edema attacks of various body
parts, most commonly edema of arms, legs, facial area or genitals, abdominal
attacks with severe pain or asphyxia due to edema in the laryngeal area.
Recemtly new treatment modalities have been developed and for a proper clinical
evaluation of these new options, a proper instrument for the interpretation of
research results is required.
.
Study objective
The objective of the study is to estimate the utility and the minimally
clinically significant symptom difference in VAS and verbal descriptor scores
as the basis for the onset of relief for skin swelling, skin pain or abdominal
pain in patients with hereditary angio-edema experiencing an acute attack.
Study design
Patients will be asked to fill in sequential VAS scores and short
questionnaires during an acute angio-edem attack (48 hrs).
Study burden and risks
The only burden is the recording of VAS scores and synmptom scores during 48
hrs after initiation of an angio-edema attack.
Invalidenstrasse 130
10115 Berlin
Duitsland
Invalidenstrasse 130
10115 Berlin
Duitsland
Listed location countries
Age
Inclusion criteria
Patients with documented type I or type II hereditary angio-edema (confirmed by medical history and C1-INH deficiency)
History of angio-edema attacks involving skin and/or abdomen
Male or Female
Age > 18 yrs
Signed written informed consent
Exclusion criteria
Diagnosis of angio-edema other than HAE, e.g. acquired angio-edema
Patients participating in other trials
Mental condition rendering the subject unable to understand the nature, scope, and possible consequences of the study
Patients unlikely to comply with protocol, e.g. uncooperative attitude, inability to return for follow-up, and unlikely to complete the study for any reason
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL13462.018.06 |