NCT01188564

Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks

A Phase III Randomized, Double-blind, Placebo-controlled Study With an Open-label Extension Evaluating the Efficacy, Safety and Immunogenicity of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks of Angioedema in Patients With HAE

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Pharming Technologies B.V. · Industry
• Sex: All
• Age: 13 Years
• Healthy volunteers: Not accepted

Summary

This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.

Detailed description

HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor. rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE. Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms. Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	rhC1INH	One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater.
DRUG	Placebo (Saline)	One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment

Timeline

• Start date: 2011-01-01
• Primary completion: 2012-12-01
• Completion: 2013-03-01
• First posted: 2010-08-25
• Last updated: 2015-08-07
• Results posted: 2015-08-07

Locations

27 sites across 11 countries: United States, Bulgaria, Canada, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Serbia, South Africa