The study is performed to investigate the s.c. versus i.v. administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable.
The study is planned as a single centre, randomised, open-label, cross-over pharmacokinetic study.
Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P, )and than switch to the treatment not administered before.
Primary Outcome Measures:
- Individual courses of C1-inhibitor levels, from these will be derived pharmacokinetic parameters [ Time Frame: i.v. and s.c.samples: 0, 0.25, 0.5, 0.75 hours and 1, 2, 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 120 and 168 hours. ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Safety of s.c. and i.v. administration of study medication. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: |
24 |
| Study Start Date: |
September 2008 |
| Estimated Study Completion Date: |
March 2010 |
| Estimated Primary Completion Date: |
March 2010 (Final data collection date for primary outcome measure) |
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1: Active Comparator
intravenous administration of C1-Inhibitor, after the end of the first observation period (at least after 7 days), each arm switches cross-over to the alternative administration mode not investigated so far
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Drug: C1-Esterase Inhibitor
1000 I.E.
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2: Active Comparator
subcutaneous administration of C1-Inhibitor. After the end of the first observation period (at least after 7 days), each arm switches cross-over to the alternative administration mode not investigated so far.
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Drug: C1-Esterase Inhibitor
1000 I.E.
|
Patients with hereditary angioedema (HAE), suffer from recurring and mostly unforeseeable attacks of acute oedema of subcutaneous tissues of various organs. The pathophysiological correlate of this disease is a deficiency in functionally active C1-Esterase Inhibitor (C1-INH).Today, two main types of HAE are described. In HAE type I, an impaired synthesis and an elevated turnover of a normal and functional active C1-INH molecule takes place, causing reduced amounts in functionally active C1-INH. In HAE type II, normal levels of a functionally impaired C1-INH molecule are synthesized. Both defects are inherited as an autosomal dominant trait. HAE type III is limited to females and not associated with C1-INH deficiency; the pathophysiology of this type remains to be determined. Corticosteroids, antihistamines or epinephrine usually do not exert any positive effect in acute attacks caused by HAE. This is of particular importance as these types of medication are often used in case of oedema in general. In case of acute oedema in patients suffering from HAE, the intravenous administration of C1-INH concentrate (e.g., Berinert P) is the treatment of choice. The study is performed to investigate the s.c. versus i.v. administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable.
Purpose
