Clinical Trial: A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-label Exploratory Phase II Study of the Safety and Immunogenicity of Repeated "rhC1INH" Administration of 50 U/Kg in Patients With Hereditary C1 Inhibitor Deficiency ("HAE"

Brief Summary: Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").

Detailed Summary:
Sponsor: Pharming Technologies B.V.

Current Primary Outcome: HAE Attacks/Week [ Time Frame: 8 weeks ]

Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).


Original Primary Outcome: to evaluate the occurrence of HAE attacks under prophylactic administration of rC1INH (50 U/Kg, once a week)

Current Secondary Outcome: The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters. [ Time Frame: 8 weeks ]

"PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4)


Original Secondary Outcome: to evaluate the pK parameters, safety and immunogenicity on repeated administration of rC1INH

Information By: Pharming Technologies B.V.

Dates:
Date Received: February 25, 2009
Date Started: June 2009
Date Completion:
Last Updated: December 20, 2012
Last Verified: December 2012