Hereditary Angioedema
Conditions
Sponsors
Akama Hideto
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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-naive subjects only) 5. Experienced at least an average of 1 HAE attack per month during the Run-in Period
Exclusion criteria
Exclusion criteria: 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(R)) 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Number of subjects with treatment emergent adverse events (TEAEs) 2. Percentage of subjects with TEAEs 3. TEAEs rates per injection 4. TEAEs rates per subject year (Time point of evaluation: up to 45 months) | — |
Secondary
| Measure | Time frame |
|---|---|
| Long-term efficacy (time point of evaluation: up to 43 months months): 1. The time-normalized number (per month and year) of Hereditary Angioedema (HAE attacks) for the entire study 2. The percentage reduction and the number of subjects experiencing at least >= 50%, >= 70%, >= 90 or equal to 100% (attack free) reduction in the time-normalized number of HAE attacks on Treatment compared to Run-in Period 3. The time-normalized number (per month and year) of HAE attacks requiring on-demand treatment in subjects on treatment 4. The time-normalized number (per month and year) of moderate and/or severe HAE attacks in subjects on treatment 5. The number and percentage of subjects rating their response to therapy as good or excellent Long-term safety (time point of evaluation: for up to 45 months): 6. The number and percentage of subjects experiencing TEAEs 7. The number and percentage of subjects experiencing adverse events of special interest (AESIs) 8. The number and percentage of subjects experiencing TEAEs for naive C1-INH subjects only 9. The number and percentage of subjects experiencing serious adverse events (SAEs), including deaths 10. The number and percentage of subjects experiencing CSL312 induced anti-CSL312 antibodies | — |
Countries
Australia, Canada, Czech Republic, Germany, Hong Kong, Hungary, Israel, Italy, Japan, New Zealand, Russia, Spain, Taiwan, The Netherlands, UK, USA
Contacts
Public ContactHideto Akama
CSL Behring K.K.
Outcome results
None listed