A study to evaluate the clinical efficacy and safety of subcutaneously administered C1-esterase inhibitor in the prevention of hereditary angioedema.
- Conditions
- Hereditary Angioedema (HAE) type I and IIMedDRA version: 17.0Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2013-000916-10-GB
- Lead Sponsor
- CSL Behring GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 80
Run-in Period Inclusion Criteria
- Males or females aged 12 years or older
- A clinical diagnosis of hereditary angioedema type I or II
- Hereditary angioedema 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 the Screening: use of a stable regimen within 3 months of Screening, with no plans to change.
Eligibility Criteria for entering Treatment Period 1
- Laboratory confirmation of type I or type II hereditary angioedema, including C1-esterase inhibitor functional activity less than 50% AND C4 antigen level below the laboratory reference range.
- No clinically significant abnormalities as assessed using laboratory parameters.
- During participation in the run-in period, subjects must have experienced hereditary angioedema attacks that required acute treatment, medical attention or caused significant functional impairment.
Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 64
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8
Run-in period exclusion criteria
- History of clinically significant arterial or venous thrombosis, or current history of a clinically significant prothrombotic risk.
- Incurable malignancies at Screening.
- Any clinical condition that will interfere with C1-esterase inhibitor therapy.
- Clinically significant history of poor response to C1-esterase inhibitor therapy for the management of hereditary angioedema.
- Receiving therapy prohibited by the protocol including medications for hereditary angioedema prophylaxis.
- Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (ie, estrogen / progesterone containing products) within 3 months before the Screening visit.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Secondary Objective: To further characterize the clinical efficacy of the 2 doses of SC CSL830.<br>To demonstrate the safety and tolerability of SC CSL830.;Primary end point(s): - The time-normalized number of hereditary angioedema attacks.;Timepoint(s) of evaluation of this end point: During the treatment phase, up to 32 weeks.;Main Objective: To demonstrate the clinical efficacy of SC CSL830 in the prophylactic treatment of HAE.To compare the clinical efficacy of 2 doses of SC<br>CSL830.<br>The comparison of the clinical efficacy of the 2 doses of CSL830 will only be done if the clinical efficacy of SC CSL830 in the prophylactic treatment of HAE is shown to be statistically significantly better than placebo.<br>
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Percentage of subjects with = 50% reduction in the number of hereditary angioedema attacks.<br>- Number of uses of rescue medication<br>-Percentage of subjects with adverse events (AEs)<br>-Percentage of subjects with AEs or other specified safety events.<br>- Percentage of subjects experiencing solicited AEs <br>- Percentage of investigational product injections resulting in solicited AEs <br>;Timepoint(s) of evaluation of this end point: - During the treatment phase, up to 32 weeks.<br>- During the treatment phase, up to 32 weeks.<br>- Within 24 hours of C1-esterase inhibitor or placebo administration.<br>- During the treatment phase, up to 32 weeks.<br>- During the treatment phase, up to 32 weeks.<br>- During the treatment phase, up to 32 weeks.<br>