Ignite Creation Date:
2025-12-25 @ 2:12 AM
Ignite Modification Date:
2025-12-28 @ 5:15 PM
Study NCT ID:
NCT05695560
Status:
COMPLETED
Last Update Posted:
2023-12-20
First Post:
2023-01-13
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D014842', 'term': 'von Willebrand Diseases'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-02-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-12', 'completionDateStruct': {'date': '2023-11-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-12-19', 'studyFirstSubmitDate': '2023-01-13', 'studyFirstSubmitQcDate': '2023-01-13', 'lastUpdatePostDateStruct': {'date': '2023-12-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-01-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-11-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Categorized Based on Bleeding Characteristics', 'timeFrame': 'Up to approximately 9 months', 'description': 'Bleeding characteristics will include categories of bleed frequency, bleed type/location and bleed severity.'}, {'measure': 'Number of Participants Categorized by Impact on Daily Life', 'timeFrame': 'Up to approximately 9 months', 'description': '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.'}, {'measure': 'Number of Participants Categorized Based on Disease Management', 'timeFrame': 'Up to approximately 9 months', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Time (Delay) to Treatment Initiation', 'timeFrame': 'Up to approximately 9 months'}, {'measure': 'Duration of Therapy Schedule', 'timeFrame': 'Up to approximately 9 months'}, {'measure': 'Number of Participants With Change in Treatment Frequency', 'timeFrame': 'Up to approximately 9 months'}, {'measure': 'Number of Participants With Bleed Control', 'timeFrame': 'Up to approximately 9 months'}, {'measure': 'Time to Bleed Control', 'timeFrame': 'Up to approximately 9 months'}, {'measure': 'Number of Participants Who Missed Days at Work/School', 'timeFrame': 'Up to approximately 9 months', 'description': 'The number of participants who missed days at work/school will include categories for patient and caregiver.'}, {'measure': 'Duration of Inpatient and Outpatient Hospital Visits', 'timeFrame': 'Up to approximately 9 months'}, {'measure': "Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time", 'timeFrame': 'Up to approximately 9 months'}, {'measure': 'Treatment Experience Based on Number of Participants Satisfied With the Treatment', 'timeFrame': 'Up to approximately 9 months'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Drug Therapy'], 'conditions': ['Von Willebrand Disease (VWD)']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://clinicaltrials.takeda.com/study-detail/cb2df18a41df432b?idFilter=%5B%22TAK-577-4002%22%5D', 'label': 'To obtain more information on the study, click here/on this link.'}]}, 'descriptionModule': {'briefSummary': "The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada.\n\nThe 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.\n\nParticipants 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.", 'detailedDescription': "This study is a non-interventional, prospective, qualitative survey to know the unmet needs of participants living with VWD and their caregivers.\n\nThe study will enroll approximately 49 patients, taking into scope both the participant's and caregiver's perspectives, and is planned to be conducted in two phases:\n\nPhase 1: Adult Participants Phase 2: Pediatric Participants The decision to proceed with Phase 2 will be determined at the completion of Phase 1.\n\nThis multi-center trial will be conducted in Canada. The overall time for data collection in this study is approximately 9 months."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '0 Years', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Participants and caregivers of participants diagnosed with VWD in Canada.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria:\n\n* Phase 1:\n* Adult participants (age ≥18 years) with severe VWD (self-BAT ≥10)\n* 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\n* For caregivers: Current caregiver of participants with severe VWD\n* For caregivers and participants:\n\n * Fluent in English or French\n * Consent to participate in an individual phone interview and to fill self-administered questionnaires\n * Additional inclusion criteria for virtual focus groups:\n\n * Access to technology (Internet and email)\n * Consent to participate in a virtual focus group with an audio recording of the session.\n* Phase 2:\n\n * Same as above for participants pediatric participants (age \\<18 years) with severe VWD (self-PBQ score of ≥3 for at least one symptom).\n\nExclusion criteria:\n\n* Phase 1:\n* Participants or caregivers of participants who do not have severe symptoms of VWD\n* Pediatric participants (age \\<18 years)\n* Participants who have not received any treatment (on-demand, regular or situational prophylaxis) within the last five years\n* Participants who are successfully treated with desmopressin or anti-fibrinolytic medications\n* Participants with inherited or acquired hemostatic or bleeding disorders other than congenital VWD (self-reported)\n* Participants and caregivers of participants who show cognitive impairment (as assessed by the research nurse at the time of screening)\n* Participants and caregivers who are qualified as health care practitioners currently working in a health-care capacity (e.g., physician, nurse, or healthcare aid)\n* Participants or caregivers who do not reside in Canada\n* Current or past participation within the last 12 months in a clinical trial\n* Phase 2: Same as above excluding participants of ≥18 years of age'}, 'identificationModule': {'nctId': 'NCT05695560', 'briefTitle': 'A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers', 'organization': {'class': 'INDUSTRY', 'fullName': 'Takeda'}, 'officialTitle': 'Unmet Needs of Patients Living With Von Willebrand Disease and Their Caregivers: Qualitative Survey on Current Standard of Care in Canada', 'orgStudyIdInfo': {'id': 'TAK-577-4002'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Phase 1: Adult Participants', 'description': '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.', 'interventionNames': ['Other: No Intervention']}, {'label': 'Phase 2: Pediatric Participants', 'description': '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.', 'interventionNames': ['Other: No Intervention']}], 'interventions': [{'name': 'No Intervention', 'type': 'OTHER', 'description': 'As this is an observational study, no intervention will be administered.', 'armGroupLabels': ['Phase 1: Adult Participants', 'Phase 2: Pediatric Participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'H3W 1Y7', 'city': 'Montreal', 'country': 'Canada', 'facility': 'YolaRx Consultants', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Takeda'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/ourmember/takeda/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR'], 'timeFrame': 'NOTE: IPD Sharing Time Frame has not been entered.', 'ipdSharing': 'YES', 'description': "Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.", 'accessCriteria': '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.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Takeda', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}