HAE Hereditary angioedema
Conditions
Interventions
Subjects will be treated with CSL312 a minimum of 1 years.
CSL312 will be administered as 1 dose (200 mg) SC once monthly for a total
minimum of 12 doses (ie, 12 months of treatment). For CSL312-na
Sponsors
CSL Behring LLC
Eligibility
Age
12 Years to 99 Years
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-naïve 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®) 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 |
|---|---|
| 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary efficacy endpoints are: • Time-normalized number of HAE attacks • The reduction in the attack rate during the Treatment Period compared to the Run-in Period • The time-normalized number of HAE attacks requiring on-demand treatment • The time-normalized number of moderate and / or severe HAE attacks The secondary safety endpoints are: • Serious adverse events (SAEs) • Deaths • Related TEAEs • TEAEs leading to study discontinuation • TEAEs by severity • Anti-CSL312 antibodies • Laboratory findings reported as adverse events (AEs) • Adverse events of special interest (AESIs) (ie, thromboembolic events [TEEs], abnormal bleeding events, severe hypersensitivity, including anaphylaxis events) • TEAEs (for nC1-INH subjects only) The secondary endpoint for the reported assessment of response to therapy is: • Subject*s Global Assessment of Response to Therapy (SGART) | — |
Countries
Netherlands
Outcome results
None listed