A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration

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")

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").

Inclusion Criteria:

• Aged at least 18 years
• Signed informed consent
• Comfirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50% of normal, and/or proven HAE ,mutation in C1INH gene.

Exclusion Criteria:

• A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs.
• A history of allergic reactions to C1INH products or rabbit protein.
• Any reported SAE related to study drug administration (withdrawal criterium)
• Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
• A diagnosis of acquired C1INH deficiency.
• Woman of child bearing potential, pregnancy or breast-feeding
• previous treatment within the last 3 months with plasma-derived C1INH
• Any clinically significant abnormality in the routine haematology, biochemistry and urinalysis
• Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives.
• Any changes since screening that would exclude subject based on above exclusion criteria.