Open-label, phase II, single arm study to evaluate the safety, immunogenicity, pharmacokinetics and efficacy of recombinant human C1 inhibitor for the treatment of acute attacks in pediatric patients with hereditary angioedema, from 2 up to and including 13 years of age - Safety of Ruconest in 2-13 year old HAE patients

Pharming Technologies B.V.0 个研究点目标入组 20 人开始时间: 2011年7月12日最近更新: 2017年10月2日

试验速览

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•From 2 up to and including 13 years of age
•Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal)
•Signed written informed consent (parental permission) signed by the legal guardian(s)
•Clinical symptoms of an acute HAE attack
•Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred
•IS score for at least one anatomical location at the time of initial evaluation of at least 3 (moderate severity or greater) without signs of spontaneous regression
•24h or more have passed since the patient’s last study treatment
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 20
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•A diagnosis of acquired C1INH deficiency (AAE)
•A medical history of allergy to rabbits or rabbit-derived products (including rhC1INH, antisera), or positive anti-rabbit epithelium (dander) IgE test (cut off>0.35 kU/L in ImmunoCap® assay (Phadia, Sweden) or equivalent)
•Treatment with investigational drug in another clinical study in the last 30 days
•Any clinical significant abnormality in the physical examination and/or the routine laboratory assessments, that in the opinion of the Investigator makes the patient unsuitable for participation in the study
•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 patient based on above exclusion criteria.
•10 HAE attacks were previously treated with study medication.
•Suspicion for an alternate explanation of the symptoms other than an acute HAE attack.
•Use of any disallowed concomitant medication since onset of acute HAE attack

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