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

• Status: Completed
• Trial end date: 2017-07-17
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Pharming Technologies B.V.
• Treatments: Complement C1 Inhibitor Protein

Criteria

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 (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) IgE test