Clinical Trial: Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent PNH Subjects

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open Label Multi Center Study of Eculizumab in Children and Adolescents With Diagnosis of Paroxysmal Nocturnal Hemoglobinuria

Brief Summary: The purpose of this study was to determine appropriate dose regimens of eculizumab in pediatric and/or adolescent patients with paroxysmal nocturnal hemoglobinuria (PNH).

Detailed Summary:

This was an open label, multicenter study of eculizumab administered intravenously (IV) to approximately 6 to 8 pediatric and adolescent patients aged 2-17 years with PNH.

Planned duration of treatment was 12 weeks.

Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Serum trough and peak concentrations of eculizumab [ Time Frame: Week 1 ]

Original Primary Outcome: To study PK/PD of Eculizumab doses in pediatric and adolescent PNH patients [ Time Frame: 15 months ]

Current Secondary Outcome: Serum trough and peak concentrations of eculizumab [ Time Frame: Weeks 6 and 12 ]

Original Secondary Outcome: Safety and Efficacy determination of the chosen dosing regimen based on the age of the patient. [ Time Frame: 15 months ]

Information By: Alexion Pharmaceuticals

Dates:
Date Received: March 23, 2009
Date Started: May 2009
Date Completion:
Last Updated: March 9, 2017
Last Verified: March 2017