Covid 19 Clinical Trials

The purpose of this study in pediatric participants (<18 years of age) with severe hereditary von Willebrand disease (VWD) is: 1. To assess the efficacy, safety, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events 2. To assess the efficacy and safety of rVWF with ADVATE during elective or emergency surgery 3. To determine the pharmacokinetic (PK) profile of rVWF

NCT02932618 Von Willebrand Disease Biological: von Willebrand factor (Recombinant) Biological: Antihemophilic Factor (Recombinant)

MeSH:Von Willebrand Diseases

Primary Outcomes

Description: Treatment success for rVWF-treated nonsurgical bleeding episodes (using a 4-point scale: Excellent, Good, Moderate, None).

Measure: Hemostatic Efficacy

Time: Within 24 hours after the last infusion of study drug following the onset of the bleeding episode (if/when the severity and/or duration of the bleeding requires the infusion of the study drug)

Secondary Outcomes

Description: If/when the severity and/or duration of the bleeding requires the infusion of the study drug.

Measure: Number of Treated Nonsurgical Bleeding Episodes with an Efficacy Rating of 'Excellent' or 'Good'

Time: Throughout the study duration of approximately 6 years

Measure: Number of Infusions per Bleeding Episode

Time: Throughout the study duration of approximately 6 years

Measure: Number of Recombinant Von Willebrand Factor (rVWF), Units per Bleeding Episode

Time: Throughout the study duration of approximately 6 years

Measure: Number of ADVATE Units (if needed), per Bleeding Episode

Time: Throughout the study duration of approximately 6 years

Description: Assessed by by the operating surgeon, based on a 4-point ordinal scale: Excellent, Good, Moderate, None.

Measure: Elective or Emergency Surgery: Assessment of Hemostatic Efficacy - Immediately After Surgery

Time: Immediately after surgery

Description: Assessed by the Investigator (hematologist) on a 4-point ordinal scale: Excellent, Good, Moderate, None.

Measure: Elective or Emergency Surgery: Overall Assessment of Hemostatic Efficacy 24 hours After the Last Perioperative Infusion of rVWF

Time: 24 hours after last perioperative rVWF infusion

Description: Assessed by the Investigator (hematologist) on a 4-point ordinal scale: Excellent, Good, Moderate, None.

Measure: Elective or emergency surgery: Overall Assessment of Hemostatic Efficacy Day 7 post-operative

Time: Post-operative Day 7

Description: Assessed by the Investigator (hematologist) on a 4-point ordinal scale: Excellent, Good, Moderate, None.

Measure: Elective or emergency surgery: Overall Assessment of Hemostatic Efficacy Day 14 post-operative

Time: Post-operative Day 14

Measure: Incidence and Severity of Adverse Events (AEs)

Time: Throughout the study period of approximately 6 years

Measure: Incidence of Thrombotic Events

Time: Throughout the study period of approximately 6 years

Measure: Incidence of Severe Hypersensitivity Reactions

Time: Throughout the study period of approximately 6 years

Measure: Development of Neutralizing Antibodies to von Willebrand Factor (VWF) and Factor VIII (FVIII)

Time: Throughout the study period of approximately 6 years

Measure: Development of Total Binding Antibodies to von Willebrand Factor (VWF)

Time: Throughout the study period of approximately 6 years

Measure: Development of Antibodies to Chinese Hamster Ovary (CHO) Proteins, Murine Immunoglobulin G (IgG), and rFurin

Time: Throughout the study period of approximately 6 years

Measure: Area Under the Plasma Concentration/Time Curve From 0 to 96 Hours Post-Infusion (AUC0-96h)