Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease

NCT01602419 | Completed Results

• 1 other identifier: Wil-20
• Study Type: observational
• Target: 120
• Locations: 10 countries
• Sites: 24

Brief Summary

This is an observational study, hence there is no study hypothesis

Conditions

Von Willebrand Disease

Outcome Measures

Primary Outcomes (2)

• Incidence of Adverse Drug Reactions (ADRs) (%)

  Medical Dictionary for Regulatory Activities (MedDRA) primary system organ class preferred term. Incidence rate = number of patients reporting the event / number of patients \* 100

  Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185])

• Tolerability Assessment of Wilate Infusions by Reason for Administration

  Tolerability was assessed using a 3-point verbal rating scale (excellent; satisfactory; unsatisfactory) according to overall feeling during and after Wilate therapy and occurrence of ADRs. Tolerability was assessed for infusions given for on-demand and prophylactic treatment, but not for infusions administered for surgeries or for the purpose of thrombogenicity assessment. In some instances, however, investigators also recorded the tolerability of infusions given for surgical prophylaxis. For infusions administered for surgeries, only those with available tolerability assessments are presented. Wilate infusion may have been administered to a patient for more than one reason and may be included in more than one category (e.g., if a patient was under Wilate prophylaxis, they could also receive Wilate for the treatment of a bleeding episode \[BE\] or surgery or menstruation).

  During and immediately after each infusion of Wilate during the study.

Secondary Outcomes (4)

• Patient and Investigator Efficacy Analysis Assessment of the Treatment of Bleeding Episodes (BEs)

  During and immediately after treatment of each BE.

• Efficacy Analysis for the Prevention of Breakthrough Bleeds During Prophylaxis

  At the end of the study for each patient (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).

• Efficacy Analysis of Surgical Prophylaxis

  During and immediately after each surgery.

• Overall Efficacy Assessment by Patient and Physician at the End of the Treatment Period

  At the end of the study for each patient (study duration: mean [±SD]: 596 days [±336]; median [range]: 732 days [2-1185]).

Other Outcomes (20)

• Safety: Number of Exposure Days to Wilate

  Throughout the duration of each patient's participation in the study (study duration: mean [±SD]: 575 days [±326]; median [range]: 731 days [2-1185]).

• Safety: Frequency of VWF Inhibitors at Baseline and Follow up

  Optional antibody tests were performed at baseline and 3-4 days (preferable 7 days) after Wilate injection.

• Adverse Drug Reactions (ADRs)

  Optional antibody tests were performed at baseline and 3-4 days (preferable 7 days) after Wilate injection

Study Arms (1)

Patients using Wilate as standard of care treatment

This patient population is being treated with Wilate as standard of care treatment

Other: Patients using Wilate as standard of care

Interventions

Patients using Wilate as standard of care OTHER

Patients with von Willebrand Disease using Wilate for a period of 2 years.

Patients using Wilate as standard of care treatment

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

VWD patients of any gender, age, or VWD type

You may qualify if:

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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (24)

Los Angeles Biomedical Research Institute

Torrance, California, 90502, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

Location

Fundación de la Hemofilia de Salta

Salta, Argentina

Location

University of Alberta

Edmonton, Alberta, Canada

Location

Vancouver General Hospital

Vancouver, British Columbia, Canada

Location

St. John Regional Hospital

Saint John, New Brunswick, Canada

Location

Eastern Regional Health Authority

St. John's, Newfoundland and Labrador, Canada

Location

McMaster University

Hamilton, Ontario, Canada

Location

Queens University

Kingston, Ontario, K7L 3N6, Canada

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Location

St. Michael's Hospital

Toronto, Ontario, Canada

Location

Maisonneuve-Rosemont Hospital

Montreal, Quebec, H1T 2M4, Canada

Location

University Hospital Ostrava

Ostrava-Poruba, Czechia

Location

Werlhof-Institut

Hanover, Germany

Location

Centro Hospitalar Cova da Beira

Covilha, Portugal

Location

Hospital Universitario de Burgos

Burgos, 09006, Spain

Location

Hospital San Pedro de Alcantara

Cáceres, 10003, Spain

Location

Hospital Fundacion Jiminez Diaz

Madrid, 28040, Spain

Location

Skåne University Hospital

Malmo, Sweden

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Hospital Pereira Rossell

Montevideo, Uruguay

Location

Sanatorio Americano

Montevideo, Uruguay

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum Samples

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Sigurd Knaub/SVP CR&D Haematology
• Organization: Octapharma AG

Sigurd.Knaub@octapharma.com

+41 55 4512141

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2012
• First Posted: May 21, 2012
• Study Start: October 1, 2012
• Primary Completion: April 1, 2018
• Study Completion: April 1, 2018
• Last Updated: January 19, 2021
• Results First Posted: December 10, 2019

Record last verified: 2020-12

Locations

ES (3); UR (2); DE (1); CZ (1); US (4); PT (1); CA (9); SE (1); GB (1); AR (1)