An open-label exploratory Phase II study of the safety and prophylactic effect of a weekly 50 U/kg rC1INH treatment in asymptomatic patients with hereditary C1INH deficiency (HAE) - OPERA

Pharming Technologies B.V.0 个研究点目标入组 25 人开始时间: 2009年4月9日最近更新: 2012年3月19日

研究概览

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•Aged at least 18 years
•Signed written informed consent
•Confirmed 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
•The occurence of an HAE attack at least every fortnight
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range

•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 eventual previous rC1INH administration (withdrawal criterium).
•Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
•A diagnosis of acquired C1INH deficiency (exclude patients with AAE).
•Women of child bearing potential, who are (or intent to become) pregnant or breast-feeding.
•Participation in another clinical study in the last month prior to this study
•Treatment with C1INH concentrates or any blood or plasma-derived material (e.g. fresh-frozen plasma) within 7 days prior to treatment with rC1INH.
•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.
•Treatment with anticoagulants (e.g) heparin or warfarin) in the last 3 days preceding treatment with rC1INH.
•Any changes since screening that would exclude subject based on above exclusion criteria.

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