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D014842: Von Willebrand Diseases

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Correlated Drug Terms (2)


Name (Synonyms) Correlation
drug323 Antihemophilic Factor (Recombinant) Wiki 1.00
drug4807 von Willebrand factor (Recombinant) Wiki 1.00

Correlated MeSH Terms (0)


Name (Synonyms) Correlation

Correlated HPO Terms (0)


Name (Synonyms) Correlation

Clinical Trials

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There is one clinical trial.


1 A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

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
Conditions
  1. Von Willebrand Disease
Interventions
  1. Biological: von Willebrand factor (Recombinant)
  2. 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)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-inf)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Mean Residence Time (MRT)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Description: Tmax will be assessed.

Measure: Time to Reach Maximal Plasma Concentration (Tmax)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Description: Cmax will be assessed.

Measure: Maximal Plasma Concentration (Cmax)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Clearance (CL)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Incremental Recovery (IR)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: In-vivo Recovery (IVR)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Elimination Phase Half-life (T1/2)

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Volume of Distribution at Steady State (Vss) for VWF:RCo, VWF:Ag and VWF:CB

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Description: von Willebrand factor: antigen (VWF:Ag); von Willebrand factor: collagen binding capacity (VWF:CB).

Measure: Area Under the Plasma Concentration/Time Curve from 0 to 96 Hours Post-infusion (AUC0-96h) for VWF:Ag and VWF:CB

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

Measure: Area Under the Plasma Concentration/Time Curve From 0 to 96 Hours Post-infusion (AUC0-96h) for Factor VIII (FVIII) Activity

Time: Within 30 minutes prior to infusion and post infusion at: SEQUENCE 1: 1, 24, 72 hours; SEQUENCE 2: 0.25, 12, and 48 hours; SEQUENCE 3: 6, 30, and 96 hours

HPO Nodes


HPO

Alphabetical listing of all HPO terms. Navigate: Correlations   Clinical Trials


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Data processed on September 26, 2020.

An HTML report was created for each of the unique drugs, MeSH, and HPO terms associated with COVID-19 clinical trials. Each report contains a list of either the drug, the MeSH terms, or the HPO terms. All of the terms in a category are displayed on the left-hand side of the report to enable easy navigation, and the reports contain a list of correlated drugs, MeSH, and HPO terms. Further, all reports contain the details of the clinical trials in which the term is referenced. Every clinical trial report shows the mapped HPO and MeSH terms, which are also hyperlinked. Related HPO terms, with their associated genes, protein mutations, and SNPs are also referenced in the report.

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