Ignite Creation Date:
2025-12-25 @ 1:02 AM
Ignite Modification Date:
2026-01-01 @ 8:57 PM
Study NCT ID:
NCT04723693
Status:
COMPLETED
Last Update Posted:
2024-08-19
First Post:
2021-01-14
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}], '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': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'simon.fletcher@ouh.nhs.uk', 'phone': '01865 225316', 'title': 'Simon Fletcher', 'organization': 'Oxford university Hospitals NHS Foundation Trust'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Over the course of the 1 hour interview', 'eventGroups': [{'id': 'EG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members', 'description': 'Qualitative interviews\n\nQualitative Interview: A single one hour semi structured qualitative interview', 'otherNumAtRisk': 28, 'deathsNumAtRisk': 28, 'otherNumAffected': 0, 'seriousNumAtRisk': 28, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members', 'description': 'Qualitative interviews\n\nQualitative Interview: A single one hour semi structured qualitative interview'}], 'classes': [{'title': 'Bleeds', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Pain', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Treatment Burden', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'Control', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Freedom', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Missed Opperyunities', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': "To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy and see if there is any reduction in the burden of the condition.", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': '16 People with Haemophilia and inhibitors were interviewed 12 with family members 14 interviews took place online, one by telephone and one in person. 6 themes emerged: bleeds; pain; treatment burden; control; freedom (for both PwHi and family members) and missed potential.'}, {'type': 'SECONDARY', 'title': 'Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab', 'description': 'Qualitative interviews\n\nQualitative Interview: A single one hour semi structured qualitative interview'}], 'classes': [{'title': 'Treatment Burden', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Control', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Freedom', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': 'To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'People with haemophilia and inhibitors and their family members'}, {'type': 'SECONDARY', 'title': 'Treatment Expectations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members', 'description': 'Qualitative interviews\n\nQualitative Interview: A single one hour semi structured qualitative interview'}], 'classes': [{'title': 'Increased sense of opportunity on new treatment', 'categories': [{'measurements': [{'value': '26', 'groupId': 'OG000'}]}]}, {'title': 'No improvement in opportunities', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'analyses': [{'groupIds': ['OG000'], 'nonInferiorityType': 'OTHER', 'otherAnalysisDescription': 'This is a qualitative interview study and as such no statistical analysis was carried out.'}], 'paramType': 'NUMBER', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': "To understand user's expectations of emicizumab and how they see future haemophilia care (less frequent injections, impact on home storage, number of bleeds, reduced hospitalisations etc).", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'people with haemophilia and inhibitors and their family members'}, {'type': 'SECONDARY', 'title': 'Treatment Impact', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members', 'description': 'Qualitative interviews\n\nQualitative Interview: A single one hour semi structured qualitative interview'}], 'classes': [{'title': 'Increased control', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Increased freedom', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'analyses': [{'groupIds': ['OG000'], 'nonInferiorityType': 'OTHER', 'otherAnalysisDescription': 'This is a qualitative interview study and as such no statistical analysis was carried out.'}], 'paramType': 'NUMBER', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': 'To describe the impact of the change in treatment on the extended family', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members', 'description': 'Qualitative interviews\n\nQualitative Interview: A single one hour semi structured qualitative interview'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': '30 participants were recruited in total.\n\n* 16 people with haemophilia and inhibitors\n* 14 family members', 'groupId': 'FG000', 'numSubjects': '30'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': '28 participants completed the study\n\n* 16 people with haemophilia and inhibitors\n* 12 family members\n\n 2 family members withdrew prior to interview\n\nTherefore 12 dyad interviews and 4 individual interviews were carried out', 'groupId': 'FG000', 'numSubjects': '28'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}]}], 'recruitmentDetails': 'Study participants will be people with inhibitors using emicizumab (prescribed by treating clinicians) in usual clinical care. Family members of participants will also be included.', 'preAssignmentDetails': 'Individual participants and a family member (if available and consented) were interviewed as a dyad pair.\n\nIndividual participants with no available family member were interviewed on their own'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members', 'description': 'Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '19', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United Kingdom', 'denoms': [{'units': 'Participants', 'counts': [{'value': '28', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '28', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': '16 people with haemophilia and inhibitors and 12 family members'}}, 'documentSection': {'largeDocumentModule': {'noSap': True, 'largeDocs': [{'date': '2019-11-04', 'size': 532357, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-01-15T11:03', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'FAMILY_BASED'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 30}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-02-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2021-01-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-03-12', 'studyFirstSubmitDate': '2021-01-14', 'resultsFirstSubmitDate': '2021-12-10', 'studyFirstSubmitQcDate': '2021-01-20', 'lastUpdatePostDateStruct': {'date': '2024-08-19', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-03-12', 'studyFirstPostDateStruct': {'date': '2021-01-26', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-08-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-01-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities)', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': "To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy and see if there is any reduction in the burden of the condition."}], 'secondaryOutcomes': [{'measure': 'Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways)', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': 'To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.'}, {'measure': 'Treatment Expectations', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': "To understand user's expectations of emicizumab and how they see future haemophilia care (less frequent injections, impact on home storage, number of bleeds, reduced hospitalisations etc)."}, {'measure': 'Treatment Impact', 'timeFrame': 'Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & thematically analysed and reported.', 'description': 'To describe the impact of the change in treatment on the extended family'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Haemophilia, Quality of Life'], 'conditions': ['Hemophilia A With Inhibitor']}, 'referencesModule': {'references': [{'pmid': '34878209', 'type': 'BACKGROUND', 'citation': 'Fletcher S, Jenner K, Holland M, Khair K. The lived experience of a novel disruptive therapy in a group of men and boys with haemophilia A with inhibitors: Emi & Me. Health Expect. 2022 Feb;25(1):443-454. doi: 10.1111/hex.13404. Epub 2021 Dec 8.'}]}, 'descriptionModule': {'briefSummary': "This study aims to examine the real-life experience and impact of using emicizumab in a cohort of patients with haemophilia and inhibitors, who were prescribed emicizumab as part of the early access to medicine schema (EAMS),those who have been in clinical trials and those now receiving emicizumab as part of routine haemophilia care.\n\nThe Investigators also intend to capture the impact of emicizumab use on the lives of close family members (parents/carers/children/partners/siblings). Each participant and his family members will be deemed a study 'dyad'.\n\nThis is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice.\n\nThe study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and therefore offers insight into how these patients and families cope with haemophilia.", 'detailedDescription': 'Emicizumab offers patients protection from bleeding with fewer injections, this should reduce treatment burden. While patient expectation is high (as evidenced by the conversations on UK haemophilia social media sites) there are risks that patients may forget to treat and thus experience bleeds. During clinical trials participants have been monitored closely and, through questionnaire completion, have described improvement in quality of life \\[Mancuso et al, 2018, Oldenburg et al 2019\\].\n\nWhat is also apparent to clinical teams is the dramatic change to life experience of not just the patient but his family - the ability to travel, to plan attending events knowing that bleeds will not occur, even potentially to have another child because of the reduction in treatment burden, particularly the time needed for treatment and rehabilitation.\n\nThis therapy represents a substantial shift in the entire life experience of living with and managing haemophilia with inhibitors. As such, there is a need to look beyond the quantitative data collected in clinical trials and to assess the real impact of therapy on the everyday lives of patients and their families, gathered using qualitative research techniques.\n\nEmicizumab has been available in the UK in clinical trials and as part of an Early Access to Medicines Scheme (EAMS) for non-trial eligible patients since spring 2018. It was recently licensed in the UK and is now available for routine clinical care for any person with haemophilia A and an inhibitor.\n\nWe hypothesise that patients and their carers/families will be excited about this new treatment option and that for most, if not all, the promise of improved care and quality of life will be a reality.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '100 Years', 'minimumAge': '8 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Male participants with Hameophilia A and inhibitors currently on emicizumab prophylaxis and a member of their family', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males\n* A diagnosis of Haemophilia A with inhibitors\n* Using emicizumab (prescribed by treating clinicians) in usual clinical care.\n\nExclusion Criteria:\n\n* No history of a Factor VIII inhibitor\n* Not currently being treated with emicizumab,\n* Does not speak English (for the interviews)\n* Does not consent to take part.'}, 'identificationModule': {'nctId': 'NCT04723693', 'acronym': 'EmiandMe', 'briefTitle': 'An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families', 'organization': {'class': 'OTHER', 'fullName': 'Haemnet'}, 'officialTitle': 'An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families', 'orgStudyIdInfo': {'id': 'v4 4Nov2019'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'individuals With Haemophilia A and with inhibitors on emicizumab', 'description': 'Qualitative interviews', 'interventionNames': ['Other: Qualitative Interview']}], 'interventions': [{'name': 'Qualitative Interview', 'type': 'OTHER', 'description': 'A single one hour semi structured qualitative interview', 'armGroupLabels': ['individuals With Haemophilia A and with inhibitors on emicizumab']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'OX3 7LE', 'city': 'Oxford', 'state': 'Oxfordshire', 'country': 'United Kingdom', 'facility': 'Oxford University Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}], 'overallOfficials': [{'name': 'Simon P Fletcher, MA', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Researcher'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Haemnet', 'class': 'OTHER'}, 'collaborators': [{'name': 'Roche Chugai', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}