A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers

NCT05695560 | Completed

• 1 other identifier: TAK-577-4002
• Study Type: observational
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada. The survey is planned to be done in two phases: The first phase will be directed at adult participants; the second phase will focus on children and teenagers. At the end of the first phase the Sponsor will decide if the second phase will be started. Participants and their caregivers will be asked to answer a set of questions either using an online questionnaire or through interviews. The participant/caregiver's perception, experience, satisfaction, and unmet needs, and need for new treatments or new indications will be determined based on their responses to the questions.

Conditions

Von Willebrand Disease (VWD)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (3)

• Number of Participants Categorized Based on Bleeding Characteristics

  Bleeding characteristics will include categories of bleed frequency, bleed type/location and bleed severity.

  Up to approximately 9 months

• Number of Participants Categorized by Impact on Daily Life

  The age-adapted impact on daily life will be categorized by questions related to quality of life, physical activity, professional life, school, financial impact, mental health, relationships, avoidance of social and physical activities, and impact on daily activities.

  Up to approximately 9 months

• Number of Participants Categorized Based on Disease Management

  Disease management will be categorized by need for subsequent therapies, need for additional investigations, time needed for disease management, impact on future planning and treatment access for aging participants.

  Up to approximately 9 months

Secondary Outcomes (9)

• Time (Delay) to Treatment Initiation

  Up to approximately 9 months

• Duration of Therapy Schedule

  Up to approximately 9 months

• Number of Participants With Change in Treatment Frequency

  Up to approximately 9 months

• Number of Participants With Bleed Control

  Up to approximately 9 months

• Time to Bleed Control

  Up to approximately 9 months

Study Arms (2)

Phase 1: Adult Participants

Adult participants with severe VWD (self-bleeding assessment tool \[BAT\] score ≥10) and their caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform.

Other: No Intervention

Phase 2: Pediatric Participants

Pediatric participants with severe VWD (self-pediatric bleeding questionnaire \[PBQ\] score of ≥3 for at least one symptom and caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform. The decision to proceed with Phase 2 will be determined following completion of Phase 1.

Other: No Intervention

Interventions

No Intervention OTHER

As this is an observational study, no intervention will be administered.

Phase 1: Adult Participants; Phase 2: Pediatric Participants

Eligibility Criteria

• Age: 0 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Participants and caregivers of participants diagnosed with VWD in Canada.

You may qualify if:

• Phase 1:
• Adult participants (age ≥18 years) with severe VWD (self-BAT ≥10)
• Participants who have received von Willebrand factor (VWF) treatment, either for on-demand treatment, regular prophylaxis, or situational prophylaxis (e.g., surgery) within the last 5 years
• For caregivers: Current caregiver of participants with severe VWD
• For caregivers and participants:
• Fluent in English or French
• Consent to participate in an individual phone interview and to fill self-administered questionnaires
• Access to technology (Internet and email)
• Consent to participate in a virtual focus group with an audio recording of the session.
• Phase 2:
• Same as above for participants pediatric participants (age \<18 years) with severe VWD (self-PBQ score of ≥3 for at least one symptom).

You may not qualify if:

• Phase 1:
• Participants or caregivers of participants who do not have severe symptoms of VWD
• Pediatric participants (age \<18 years)
• Participants who have not received any treatment (on-demand, regular or situational prophylaxis) within the last five years
• Participants who are successfully treated with desmopressin or anti-fibrinolytic medications
• Participants with inherited or acquired hemostatic or bleeding disorders other than congenital VWD (self-reported)
• Participants and caregivers of participants who show cognitive impairment (as assessed by the research nurse at the time of screening)
• Participants and caregivers who are qualified as health care practitioners currently working in a health-care capacity (e.g., physician, nurse, or healthcare aid)
• Participants or caregivers who do not reside in Canada
• Current or past participation within the last 12 months in a clinical trial
• Phase 2: Same as above excluding participants of ≥18 years of age

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

YolaRx Consultants

Montreal, H3W 1Y7, Canada

Location

Related Links

• To obtain more information on the study, click here/on this link.

MeSH Terms

Conditions

von Willebrand Diseases

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

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: January 13, 2023
• First Posted: January 25, 2023
• Study Start: February 24, 2023
• Primary Completion: November 30, 2023
• Study Completion: November 30, 2023
• Last Updated: December 20, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: NOTE: IPD Sharing Time Frame has not been entered.
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

CA (1)