ong-term safety and efficacy of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema attacks
- Conditions
- Hereditary angioedemaMedDRA version: 23.1Level: 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
- EUCTR2020-003918-12-NL
- Lead Sponsor
- CSL Behring LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 150
1. Males and females aged = 12 years
2. Diagnosed with clinically confirmed C1-INH HAE
3. Experienced = 3 HAE attacks during the 3 months before Screening
4. Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only)
5. Experienced at least an average of 1 HAE attack per month during the Run-in Period
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 110
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20
1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria
2. Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period
3. Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period.
4. Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening
5. Female or male subjects who are fertile and sexually active not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of the last dose of CSL312
6. Pregnant, breastfeeding, or not willing to cease breastfeeding
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of the study is to evaluate the long-term safety of SC administration of CSL312 in the prophylactic treatment of subjects with C1-INH HAE<br>;Secondary Objective: The secondary objectives of this study are to evaluate the long-term efficacy, safety and patient reported assessment of response to therapy<br>;Primary end point(s): 1. Number of subjects with treatment emergent adverse events (TEAEs)<br>2. Percentage of subjects with TEAEs<br>3. TEAEs rates per injection<br>4. TEAEs rates per subject year<br>;Timepoint(s) of evaluation of this end point: 1. Up to 45 months<br>2. Up to 45 months<br>3. Up to 45 months<br>4. Up to 45 months<br><br><br>
- Secondary Outcome Measures
Name Time Method