Clinical trial NCT01602419: Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
Official title: Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Von Willebrand Disease
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2012-05-21
Enrollment: 120
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: VWD patients of any gender, age, or VWD type

Brief summary: This is an observational study, hence there is no study hypothesis


Eligibility criteria: Inclusion Criteria:

* Patients with a diagnosis of von Willebrand Disease who have been prescribed Wilate

Primary outcomes:
measure: Incidence of Adverse Drug Reactions (ADRs) (%)
timeFrame: Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185])
measure: Tolerability Assessment of Wilate Infusions by Reason for Administration
timeFrame: During and immediately after each infusion of Wilate during the study.
Secondary outcomes:
measure: Patient and Investigator Efficacy Analysis Assessment of the Treatment of Bleeding Episodes (BEs)
timeFrame: During and immediately after treatment of each BE.
measure: Efficacy Analysis for the Prevention of Breakthrough Bleeds During Prophylaxis
timeFrame: At the end of the study for each patient (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
measure: Efficacy Analysis of Surgical Prophylaxis
timeFrame: During and immediately after each surgery.
measure: Overall Efficacy Assessment by Patient and Physician at the End of the Treatment Period
timeFrame: At the end of the study for each patient (study duration: mean [±SD]: 596 days [±336]; median [range]: 732 days [2-1185]).
Other outcomes:
measure: Safety: Number of Exposure Days to Wilate
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 575 days [±326]; median [range]: 731 days [2-1185]).
measure: Safety: Frequency of VWF Inhibitors at Baseline and Follow up
timeFrame: Optional antibody tests were performed at baseline and 3-4 days (preferable 7 days) after Wilate injection.
measure: Adverse Drug Reactions (ADRs)
timeFrame: Optional antibody tests were performed at baseline and 3-4 days (preferable 7 days) after Wilate injection
measure: Safety: Patients With Thrombogenicity Values >2 Times the Upper Limit of Normal (ULN)
timeFrame: Optional thrombogenicity tests were performed at baseline and 1, 3 and 24 hours after each administration of Wilate.
measure: Wilate Dosage Per Infusion for the Treatment of Acute Bleeding Episodes (BEs; On-demand)
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 677 days [±264]; median [range]: 749 days [40-1052]).
measure: Wilate Dosage for the Treatment of Acute Bleeding Episodes (BEs; On-demand) Per Bleeding Episode (BE)
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 677 days [±264]; median [range]: 749 days [40-1052]).
measure: Wilate Doses for the Treatment of Menstrual Bleeding Episodes (BEs)
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 553 days [±296]; median [range]: 713 days [125-840]).
measure: Exposure Days for Prophylactic Treatment With Wilate
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 761 days [±251]; median [range]: 773 days [144-1185]).
measure: Number of Infusions of Prophylactic Treatment With Wilate Per Week
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 761 days [±251]; median [range]: 773 days [144-1185]).
measure: Dosage for Prophylactic Treatment With Wilate Per Week
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 761 days [±251]; median [range]: 773 days [144-1185]).
measure: Dosage for Prophylactic Treatment With Wilate Per Infusion
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 761 days [±251]; median [range]: 773 days [144-1185]).
measure: Exposure Days for Prophylactic Wilate Treatment on a Continuous Basis
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
measure: Number of Infusions Per Week for Prophylactic Wilate Treatment on a Continuous Basis
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
measure: Dosage for Prophylactic Wilate Treatment on a Continuous Basis
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
measure: Dosage for Prophylactic Wilate Treatment Per Infusion on a Continuous Basis
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
measure: Dosage of Wilate Per Infusion for the Treatment of Breakthrough Bleeds
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 561 days [±251]; median [range]: 773 days [144-1185]).
measure: Dosage of Wilate for the Treatment of Breakthrough Bleeds Per Breakthrough Bleed
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 561 days [±251]; median [range]: 773 days [144-1185]).
measure: Wilate Dosage Per Infusion for the Prevention of Bleeding During and After Surgery
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 568 days [±349]; median [range]: 732 days [2-1185]).
measure: Wilate Dosage Per Procedure for the Prevention of Bleeding During and After Surgery
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 568 days [±349]; median [range]: 732 days [2-1185]).
measure: Number of Patients With Breakthrough Bleeds During Prophylaxis
timeFrame: Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 761 days [±251]; median [range]: 773 days [144-1185]).
Locations:
city: Torrance
country: United States
facility: Los Angeles Biomedical Research Institute
state: California
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Salta
country: Argentina
facility: Fundación de la Hemofilia de Salta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: Vancouver General Hospital
state: British Columbia
city: Saint John
country: Canada
facility: St. John Regional Hospital
state: New Brunswick
city: St. John's
country: Canada
facility: Eastern Regional Health Authority
state: Newfoundland and Labrador
city: Hamilton
country: Canada
facility: McMaster University
state: Ontario
city: Kingston
country: Canada
facility: Queens University
state: Ontario
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Montreal
country: Canada
facility: Maisonneuve-Rosemont Hospital
state: Quebec
city: Ostrava-Poruba
country: Czechia
facility: University Hospital Ostrava
city: Hannover
country: Germany
facility: Werlhof-Institut
city: Covilhã
country: Portugal
facility: Centro Hospitalar Cova da Beira
city: Burgos
country: Spain
facility: Hospital Universitario de Burgos
city: Cáceres
country: Spain
facility: Hospital San Pedro de Alcantara
city: Madrid
country: Spain
facility: Hospital Fundacion Jiminez Diaz
city: Malmö
country: Sweden
facility: Skåne University Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Montevideo
country: Uruguay
facility: Hospital Pereira Rossell
city: Montevideo
country: Uruguay
facility: Sanatorio Americano
Study officials:
References: