Efficacy and safety of CSL830 in hereditary angioedema
Research type
Research Study
Full title
A double-blind, randomized, placebo-controlled, cross-over study to evaluate the clinical efficacy and safety of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema
IRAS ID
136905
Contact name
Hilary Longhurst
Contact email
Sponsor organisation
CSL Behring GmbH (CSL)
Eudract number
2013-000916-10
ISRCTN Number
N/A
Research summary
Hereditary angioedema (HAE) is an inherited disease that can cause episodes of swelling of the skin and tissues of the abdomen, neck, throat, face, arms and legs. In most cases, the disease is due to the lack of a working protein called C1-esterase inhibitor (C1-INH). Therapy for HAE includes giving C1-INH to treat an episode of swelling, or to give it regularly to prevent these episodes from occurring. However, C1-INH currently has to be given directly into a vein and this is not practical for some patients. A more convenient way of giving C1-INH by an injection under the surface of the skin is being developed. This form of C1-INH is called CSL830 (the study medicine).
CSL830 is an experimental medicine which means it has not been approved by the drug approval agency.
In this study, we will test how well CSL830 helps prevent episodes of swelling due to HAE.
This study will have two treatment periods that will each last 16 weeks. Participants will get one of two doses of CSL830 for one treatment period and will get a placebo for the other treatment period. Placebo does not contain any medicine. Participants will inject CSL830 and placebo under the skin in the abdomen using a needle twice per week.
If they need it, participants will also be able to use rescue medicine to help treat any episodes of swelling associated with HAE during the study. The Sponsor can supply the rescue medicine called Berinert.
Berinert contains the same medicine as CSL830, but is given by an injection to the vein.
About 100 participants with HAE will be in the study, they will be in the study for up to 45 weeks.REC name
London - Riverside Research Ethics Committee
REC reference
13/LO/1795
Date of REC Opinion
10 Dec 2013
REC opinion
Favourable Opinion