Hereditary Angioedema Type I Clinical Trial
Official title:
Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema
A multicentre study to investigate pharmacokinetics, clinical efficacy and safety of
nanofiltered Cetor® (called C1-esteraseremmer-N during the development phase) for the
treatment of hereditary angioedema (HAE) will be performed. This study KB2003.01 consists of
three parts, part A pharmacokinetics (phase II), part B treatment of attacks of angioedema
(phase III) and part C prophylactic use of C1 inhibitor (phase III). Part B + C will provide
data on the efficacy of C1-esteraseremmer-N.
The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the
currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B
immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide
more safety regarding viruses.
In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary
angioedema will be compared with the current registered product, Cetor®, in a randomised,
blinded cross-over design. This study has to provide evidence that changes in the
manufacturing process have not affected pharmacokinetics. In addition, this study provides
data on safety of C1-esteraseremmer-N.
A multicentre study to investigate pharmacokinetics, clinical efficacy and safety of
nanofiltered Cetor® (called C1-esteraseremmer-N during the development phase) for the
treatment of hereditary angioedema (HAE) will be performed. This study KB2003.01 consists of
three parts, part A pharmacokinetics (phase II), part B treatment of attacks of angioedema
(phase III) and part C prophylactic use of C1 inhibitor (phase III). Part B + C will provide
data on the efficacy of C1-esteraseremmer-N.
The changes in the manufacturing process of C1-esteraseremmer-N, compared to Cetor® (the
currently marketed C1-inhibitor product), nanofiltration and omission of hepatitis B
immunoglobulin, most likely will not affect tolerability. The nanofiltration will provide
more safety regarding viruses.
In part A, the pharmacokinetics of C1-esteraseremmer-N in patients with hereditary
angioedema will be compared with the current registered product, Cetor®, in a randomised,
blinded cross-over design. This study has to provide evidence that changes in the
manufacturing process have not affected pharmacokinetics. In addition, this study provides
data on safety of C1-esteraseremmer-N. Twelve HAE patients without signs of an attack will
receive an administration of 1,000 U, 1,500 U or 2,000 U of C1-esteraseremmer-N or Cetor®
and later on the same dose of the other product. Both antigenic and functional C1 inhibitor
levels will be determined. Laboratory safety parameters and adverse events will be monitored
as well
;
Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Prevention
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