Evaluation of the Pharmacokinetic Profile, Clinical Efficacy and Safety of the Von Willebrand Factor Contained in FANHDI® (Double-inactivated Human Anti-hemophilic Factor) in Pediatric Subjects With Severe Von Willebrand Disease

NCT02472665 Phase 4 RECRUITING

Multicenter, prospective, non-controlled study in a pediatric cohort (\<6 years-old) with severe (type 2 or 3) hereditary Von Willebrand Disease (VWD).

Details

Lead sponsor	Grifols Therapeutics LLC
Phase	Phase 4
Status	RECRUITING
Enrolment	8
Start date	2013-12
Completion	2026-12

Conditions

• Von Willebrand Disease

Interventions

• plasma-derived FVIII/VWF concentrate

Primary outcomes

• AUC^0-inf of coagulation factor VIII activity (FVIII:C) — Prior to the first infusion up to 72 hours postinfusion
  Cumulative area under the concentration time curve extrapolated to infinity of FVIII:C
• AUC^0-inf of von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) — Prior to the first infusion up to 72 hours postinfusion
  Cumulative area under the concentration time curve extrapolated to infinity of VWF:RCo
• AUC^0-inf of von Willebrand factor antigen (VWF:Ag) — Prior to the first infusion up to 72 hours postinfusion
  Cumulative area under the concentration time curve extrapolated to infinity of VWF:Ag
• AUC^0-inf of von Willebrand factor: Collagen binding activity (VWF:CB) — Prior to the first infusion up to 72 hours postinfusion
  Cumulative area under the concentration time curve extrapolated to infinity of VWF:CB
• AUC^0-T of FVIII:C — Prior to the first infusion up to 72 hours postinfusion
  Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of FVIII:C
• AUC^0-T of VWF:RCo — Prior to the first infusion up to 72 hours postinfusion
  Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:RCo

Countries

Spain