Hereditary angioedema (HAE) is characterized by congenital C1-INH deficiency. This is reflected by insufficient plasma concentrations of C1 – inhibitor or by synthesis of dysfunctional C1 – inhibitor molecules, which can lead to attacks of abdominal pain and/or severe swelling in the tissues e.g. of the face. Replacement with C1 esterase inhibitor concentrate is the treatment of choice of an acute attack of HAE. MedDRA version: 7.0 Level: LLT Classification code 10019860
Conditions
Interventions
Trade Name: Berinert® P
Product Name: C1 esterase inhibitor concentrate
Product Code: CE1145
Pharmaceutical Form: Powder and solvent for solution for infusion
Current Sponsor code: CE1145
Other descri
Sponsors
ZLB Behring GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male and female subjects >= 6 years - Documented congenital C1-INH deficiency with functional or immunogenic C1-INH level and C4 antigen level below the lower limit of the local laboratory reference range - Documented history of abdominal or facial attacks - Acute moderate/severe abdominal or facial attack with a duration not longer than 5.0 hours at the time of study medication administration - Informed written consent has been obtained (signed by subject, or, in case of a minor, by his/her legally acceptable representative(s)) Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Life expectancy 40 years, no family history, no known HAE mutation) - Abdominal pain caused by other pathology (e. g., appendicitis, myocardial infarction) - End-stage liver disease (i.e., Child-Pugh-score B or C) - HIV positive - Fever (> 38.3°C) - High white blood cell count (> 20.0 x 109/L ) - Pregnant women (pregnancy test required), women currently breast-feeding, or with the intention to breast feed - Treatment with any other investigational drug in the last 30 days before study entry - Treatment with any C1-INH concentrate within the previous 7 days - Treatment with fresh frozen plasma or native plasma within the previous 7 days - Treatment with ACE inhibitors within the previous 4 weeks - Narcotic pain medication and/or anti-emetics between start of attack and enrollment (signing of informed consent) - Evidence of narcotic seeking behavior and/or drug addiction (including alcohol abuse) - Mental condition rendering the subject (or the subject’s legally acceptable representative(s)) unable to understand the nature, scope and possible consequences of the study - Prior inclusion into this study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To show that pasteurized C1-INH concentrate (Berinert® P) shortens the time to onset of relief of symptoms of abdominal or facial HAE attack compared to placebo ;Secondary Objective: - To compare the efficacy of two different dosing schemes of Berinert® P in abdominal or facial attacks of edema due to HAE - To compare the safety of Berinert® P in subjects with HAE between the three treatment groups;Primary end point(s): Time to onset of relief from the evaluated (abdominal or facial) attack will be assessed by the investigator every 15 min for the first 2 hours, every 30 min for the next 2 hours, and at 5, 6, 7, 8, 12, 16, 20 and 24 hours after administration of the study medication. The investigator or a delegate will ask each subject at the above defined time points: 1. Taking into account all of the symptoms you experienced with this HAE attack, are you confident that it is starting to improve? If the answer to Question 1 has been “yes” for two consecutive time points, asking Question 1 will stop. The time of onset of relief from attack is defined by the time determined at the first of the two consecutive “yes” responses. If rescue medication or non-permitted medication was administered before this time has been reached, the subject will be considered as a non-responder and the time to onset of relief will be set to a poor/failure outcome for the primary analysis. Subjects will not be discharged from hospital, before the relief from symptoms of the evaluated (abdominal or facial) attack has started, including both the time to reaching the primary endpoint and the time to relief after rescue medication, if applicable. In addition, the 4-hour sample for C1-INH and C4 determination is to be taken before discharge. | — |
Countries
Bulgaria, Czech Republic, Hungary, Spain, Sweden, United Kingdom
Outcome results
None listed