NCT06361537

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 124 (estimated)
• Sponsor: Octapharma · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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

Conditions

• Acute Hereditary Angio Edema

Interventions

Type	Name	Description
DRUG	OCTA-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)
OTHER	Placebo	0.1 mL/kg BW 0.9% sodium chloride injection

Timeline

• Start date: 2024-04-30
• Primary completion: 2026-12-01
• Completion: 2027-06-01
• First posted: 2024-04-12
• Last updated: 2025-10-03

Locations

22 sites across 12 countries: United States, Albania, Argentina, Armenia, Bulgaria, Mexico, Montenegro, Peru, Romania, Serbia, Turkey (Türkiye), Ukraine

Regulatory

• FDA-regulated drug study