A study to evaluate the long-term clinical safety and efficacy of subcutaneously administered C1-esterase inhibitor in the prevention of hereditary angioedema
- Conditions
- MedDRA version: 17.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disordersHereditary Angioedema Types I and IITherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2014-001054-42-GB
- Lead Sponsor
- CSL Behring GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 126
• Males or females aged 6 years or older.
• A confirmed diagnosis of HAE type I or II.
• HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
• For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit.
Are the trial subjects under 18? yes
Number of subjects for this age range: 11
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 88
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 11
• Incurable malignancies.
• Any clinical condition that will interfere with the evaluation of C1-INH therapy.
• Clinically significant history of poor response to C1-esterase therapy for the management of HAE.
• Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.
• Inability to have HAE managed pharmacologically with on-demand treatment.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <br> Main Objective: To assess the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.<br> ;<br> Secondary Objective: • To further characterize the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.<br> <br> • To characterize the clinical efficacy of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.<br> ;<br> Primary end point(s): The person-time incidence rates of specified safety events.<br> ;<br> Timepoint(s) of evaluation of this end point: During the treatment phase, up to 52 weeks.<br>
- Secondary Outcome Measures
Name Time Method