Prospective, Multicenter, Randomized, Double-blind, Parallel Group, Placebo- Controlled, Efficacy and Safety Phase 3 Study of an Intravenous Human Plasma- Derived C1 Esterase Inhibitor (C1-INH) Concentrate in Participants With Congenital C1-INH Deficiency for the Treatment and Pre-procedure Prevention of Acute Hereditary Angioedema Attacks
Octapharma
Summary
Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks
Eligibility
- Age range
- 2+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Is at least 18 years of age (applicable for 1st study phase) or is at least 2 years of age (applicable for 2nd study phase) 2. Has confirmed diagnosis of HAE type I or II 3. Has had at least 3 moderate or severe HAE attacks (excluding extremity attacks) in the last 3 months before the Screening Visit. For participants ≥2 and ≤12 years of age, has had at least 1 moderate or severe HAE attack (excluding extremity attacks) in the last 6 months before Screening Visit 4. Has a documented congenital C1-INH functional activity \<50% with or without C1-INH deficiency and C4 ant…
Interventions
- DrugOCTA-C1-INH
OCTA-C1-INH is a stable, sterile, virus-inactivated, nano-filtered, highly purified concentrate of human C1-INH prepared from pooled human plasma. After reconstitution in 2.5mL water for injection, the solution can be administered as a slow IV injection. OCTA-C1-INH is given as a dose of 20 IU/kg body weight (BW)
- OtherPlacebo
0.1 mL/kg BW 0.9% sodium chloride injection
Locations (22)
- Octapharma Research SiteCentennial, Colorado
- Octapharma Research SiteFarmington Hills, Michigan
- Octapharma Research SiteToledo, Ohio
- Octapharma Research SiteTirana
- Octapharma Research SiteRosario
- Octapharma Research SiteYerevan