A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema
Hereditary Angioedema Types I and II
About this trial
This is an interventional prevention trial for Hereditary Angioedema Types I and II focused on measuring Hereditary Angioedema
Eligibility Criteria
Inclusion Criteria:
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 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, required medical attention, or caused significant functional impairment.
Exclusion Criteria:
Run-In Period Exclusion Criteria:
- History of clinical 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 the evaluation of C1-esterase inhibitor therapy.
- Clinically significant history of poor response to C1-esterase 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 (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit.
Sites / Locations
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Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Experimental
Experimental
Experimental
Higher-volume placebo, then low-volume C1-esterase inhibitor
Low-volume C1-esterase inhibitor, then higher-volume placebo
Low-volume placebo, then higher-volume C1-esterase inhibitor
Higher-volume C1-esterase inhibitor, then low-volume placebo
A higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks.
A low-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a higher-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks.
A low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks then a higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks.
A higher-volume dose of C1-esterase inhibitor will be administered subcutaneously twice a week for up to 16 weeks, then a low-volume dose of placebo will be administered subcutaneously twice a week for up to 16 weeks.