NCT02472665

Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease

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

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Grifols Therapeutics LLC · Industry
• Sex: All
• Age: 2 Months – 6 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a multicenter, prospective, open-label, and single-arm study. The study population is planned to include 8 pediatric subjects (\<6 years of age) with severe (type 2 or 3) hereditary VWD without inhibitors and with no active bleeding at the time of inclusion. Eligible subjects will receive a single dose of Fanhdi for a PK evaluation and will be followed for 12 months for which the efficacy and safety of Fanhdi will be assessed. In addition, the type 3 VWD subjects, after 6 months of follow-up of the first infusion, will receive the second dose as in the 1st PK evaluation and undergo a 2nd PK evaluation. The study will consist of 2 phases: * PK profile evaluation in which all eligible subjects will receive a single dose of 80 IU/kg von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) of Fanhdi. In addition, after 6 months of follow-up of the first infusion, type 3 VWD subjects will receive the second dose of Fanhdi and undergo a 2nd PK evaluation with a reduced sampling schedule. * A 12-month Follow-up period during which the safety and efficacy of Fanhdi will be assessed in the prevention and management of bleeding episodes and/or management of perioperative hemostasis during surgery and/or invasive procedures.

Conditions

• Von Willebrand Disease

Interventions

Type	Name	Description
DRUG	plasma-derived FVIII/VWF concentrate	1 single dose of 80 IU/kg VWF:RCo of Fanhdi will be administered

Timeline

• Start date: 2013-12-01
• Primary completion: 2025-04-01
• Completion: 2026-12-01
• First posted: 2015-06-16
• Last updated: 2024-07-08

Locations

4 sites across 1 country: Spain