Safety and efficacy of C1 esterase inhibitor in prophylactic treatment
Research type
Research Study
Full title
An open-label, randomized study to evaluate the long-term clinical safety and efficacy of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of hereditary angioedema
IRAS ID
164715
Contact name
Hilary Longhurst
Contact email
Sponsor organisation
CSL Behring GmbH
Eudract number
2014-001054-42
Duration of Study in the UK
1 years, 7 months, 0 days
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 (C1INH). Therapy for HAE includes giving C1INH to treat an episode of swelling, or to give it regularly to prevent these episodes from occurring. However, C1INH currently has to be given directly into a vein and this is not practical for some patients. A more convenient way of giving C1INH by an injection under the surface of the skin is being developed. This form of C1INH 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; Treatment Period 1 (24 weeks) will be a fixed dose period. Treatment Period 2 (28 weeks) will be a dose adjustment period to allow for individual optimization of prophylaxis. Participants will inject CSL830 under the skin in the abdomen using a needle twice per week. If they need it, participants will also be able to use on-demand medicine to help treat any episodes of swelling associated with HAE during the study.
About 110 participants with HAE will be in the study, they will be in the study for up to 58 weeks.REC name
London - Hampstead Research Ethics Committee
REC reference
14/LO/2200
Date of REC Opinion
9 Jan 2015
REC opinion
Further Information Favourable Opinion