Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients

Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of rhC1INH for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2-13 Years of Age

This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.

This study was an open-label, Phase 2, non-comparative, multinational, multicenter clinical study in pediatric patients from 2 up to and including 13 years of age, with a confirmed diagnosis of HAE. Patients were eligible for treatment with recombinant human C1-inhibitor (rhC1INH) if they presented to the clinic within 5 hours of onset with an acute attack of at least moderate severity without signs of spontaneous regression. Patients received rhC1INH at a dose of 50 U/kg body weight up to a maximum of 4200 U. The reconstituted solution was administered as a slow intravenous (iv) injection over approximately 5 minutes. The patients remained in hospital and were closely monitored in the study center for at least 4 hours after study medication administration.

Inclusion Criteria:

• 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 Form (ICF)(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
• Attack severity moderate or greater, as rated by the investigator

Exclusion Criteria:

• A diagnosis of acquired C1INH deficiency (AAE)
• A medical history of allergy to rabbits or rabbit-derived products or positive anti-rabbit epithelium (dander) immunoglobuline E (IgE) test