Treatment of hereditary angioedema with nanofiltered C1-esterase inhibitor concentrate (Cetor®): multi-center phase II and III studies to assess pharmacokinetics, clinical efficacy and safety.


Abstract

From 1997, plasma-derived C1-inhibitor concentrate (Cetor®) has been available to HAE and AAE patients. Recently, a virus reducing 15 nm nanofiltration step has been introduced in the production process. A randomized, double-blind controlled cross-over study was performed to compare the pharmacokinetics (PK) of nanofiltered (C1-INH-NF) with conventional C1-inhibitor (C1-INH). Efficacy and safety were investigated in an open-label, on-demand and a prophylactic study.

No differences in pharmacokinetic parameters between C1-INH and C1-INH-NF were found (13 non-symptomatic HAE patients). Both C1-inhibitor products equally increased plasma C4 levels.

In the on-demand study, 14 acute angioedema attacks in 8 patients were analyzed.

In the prophylactic study, 1 AAE and 5 HAE patients experienced in total 31 attacks during 748 observation days.

In total 180,000 units of C1-INH-NF were administered.

No product-related adverse events occurred, and no anti-C1-antibodies were induced.

Nanofiltration in the production process of C1-inhibitor did not affect the pharmacokinetics, efficacy, and safety.


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Publication date

2012-02-27


Journal

Clinical immunology
Clin Immunol (1521-7035)

Journal topics


Language

Eng.


Copyright

Department of Internal Medicine, Academic Medical Centre, Amsterdam, The Netherlands.


Release reference

Clin Immunol. 2012 Mar;142(3):280-90



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