Efficacy and safety study of vWF SD-35-DH (WILFACTIN) in children under 6 years of age - NOT APPLICABLE

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2004-005051-34-BE

Enrollment

Registered

2007-05-31

Start date

2005-04-18

Completion date

Unknown

Last updated

2015-08-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Von Willebrand disease Classification code 10047715

Interventions

Trade Name: WILFACTIN Product Name: WILFACTIN Product Code: VWF SD-35-DH Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: human von Willebrand factor Concentrat

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - age =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: - positive VWF and/or FVIII inhibitors - platelets < 100 x 10exp9/l (except for type 2B patients)

Design outcomes

Primary

Measure	Time frame
Main Objective: Main objective : to evaluate, in children presenting with inherited von Willebrand factor (VWF) deficiencies, the biological and clinical efficacy of WILFACTIN for the treatment of bleeding episodes and for the prevention of haemorrhages during surgery or invasive procedures when desmopressin is ineffective or contraindicated. ;Secondary Objective: Secondary objectives : to evaluate the safety, especially the immunological safety, of WILFACTIN in a paediatric population. ;Primary end point(s): - The biological efficacy of WILFACTIN will be evaluated based on VWF recovery after administration of 100 IU/kg at inclusion of the study and without bleeding ongoing, - Efficacy of treatment for bleeding episodes will be rated at the end of the episode by the doctor, using the following scale: "excellent", "good", "moderate", "none". - Efficacy of treatment during surgery will be rated at the end of hospitalisation by the doctor, using the same scale. Blood loss and use of transfusions will be recorded and analysed in comparison to normal subject. - Efficacy of long-term prophylaxis treatment with vWF SD-35-DH will be judged by the absence of spontaneous bleeding episodes or the clear reduction of haemorrhages.	—

Measure

Time frame

Main Objective: Main objective : to evaluate, in children presenting with inherited von Willebrand factor (VWF) deficiencies, the biological and clinical efficacy of WILFACTIN for the treatment of bleeding episodes and for the prevention of haemorrhages during surgery or invasive procedures when desmopressin is ineffective or contraindicated. ;Secondary Objective: Secondary objectives : to evaluate the safety, especially the immunological safety, of WILFACTIN in a paediatric population. ;Primary end point(s): - The biological efficacy of WILFACTIN will be evaluated based on VWF recovery after administration of 100 IU/kg at inclusion of the study and without bleeding ongoing, - Efficacy of treatment for bleeding episodes will be rated at the end of the episode by the doctor, using the following scale: "excellent", "good", "moderate", "none". - Efficacy of treatment during surgery will be rated at the end of hospitalisation by the doctor, using the same scale. Blood loss and use of transfusions will be recorded and analysed in comparison to normal subject. - Efficacy of long-term prophylaxis treatment with vWF SD-35-DH will be judged by the absence of spontaneous bleeding episodes or the clear reduction of haemorrhages.

—

Countries

Belgium

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026