NCT01359969

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Pharming Technologies B.V. · Industry
• Sex: All
• Age: 2 Years – 13 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	rhC1INH	Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).

Timeline

• Start date: 2012-01-17
• Primary completion: 2017-07-17
• Completion: 2017-07-17
• First posted: 2011-05-25
• Last updated: 2024-03-29
• Results posted: 2024-03-29

Locations

16 sites across 10 countries: United States, Czechia, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Slovakia

Regulatory

• FDA-regulated drug study