NCT02093897

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

A Phase III Open-label Pharmacokinetic, Efficacy and Safety Study of rVIII-SingleChain in a Pediatric Population With Severe Hemophilia A

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 84 (actual)
• Sponsor: CSL Behring · Industry
• Sex: Male
• Age: 11 Years
• Healthy volunteers: Not accepted

Summary

This is an international, multicenter, open-label study to assess the efficacy, safety, and pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe hemophilia A. A minimum of 25 previously treated subjects ≥ 6 to \< 12 years of age and at least 25 subjects \< 6 years of age who have undergone \> 50 exposure days (EDs) with a previous Factor VIII (FVIII) product are planned to be enrolled. Subjects will be assigned to either an on-demand or prophylaxis treatment regimen and will receive rVIII-SingleChain at a dose to be determined by the investigator. Hemostatic efficacy will be assessed by the subject/caregiver and the investigator who will assess overall efficacy by a 4-point scale.

Conditions

• Congenital Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	rVIII-SingleChain

Timeline

• Start date: 2014-03-01
• Primary completion: 2015-08-01
• Completion: 2015-08-01
• First posted: 2014-03-21
• Last updated: 2017-01-27
• Results posted: 2017-01-27

Locations

37 sites across 19 countries: United States, Australia, Austria, France, Georgia, Germany, Italy, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Romania, Spain, Switzerland, Thailand, Turkey (Türkiye), Ukraine