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| Name | Class |
|---|---|
| UniQure Biopharma B.V. | INDUSTRY |
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This study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people and families affected by haemophilia who have been or will be exposed to gene therapy.
This is a prospective observational multiple cohort qualitative research study to be conducted among diverse groups within the haemophilia community whose lives may have been impacted by gene therapy.
The 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 offer insight into how these patients and families cope with haemophilia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Early dose finding cohort | People with Haemophilia who took part in the early dose finding studies |
| |
| Subsequent studies group cohort | People with haemophilia who took part in subsequent gene therapy studies |
| |
| Withdrawn/ineligible Cohort | People with haemophilia who were withdrawn or proved ineligible |
| |
| Not interested cohort | People with haemophilia who are definitely not interested in gene therapy |
| |
| Not offered cohort | People with haemophilia who are interested in gene therapy but have not been the opportunity to take part |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Qualitative interview | Other | Qualitative semi structured interview |
|
| Measure | Description | Time Frame |
|---|---|---|
| patient expectations | To explore the expectations that patients, and families in the UK have of gene therapy and its position in contemporary and future haemophilia management | Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach |
| Measure | Description | Time Frame |
|---|---|---|
| Lived experience | To understand the lived experience of people who have undergone gene therapy | Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach |
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Inclusion Criteria:
Exclusion Criteria:
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People with haemophilia and a member of their family
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| Name | Affiliation | Role |
|---|---|---|
| Simon P Fletcher | Researcher | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oxford University Hospitals NHS Foundation Trust | Oxford | Oxfordshire | OX3 7LE | United Kingdom |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 13, 2020 | Jan 20, 2021 | Prot_000.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| Impact of gene therapy | To understand the as yet "unseen" impact of gene therapy on the extended family | Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach |
| Impact of ineligibility | To understand the impact of ineligibility for gene therapy trials | Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach |
| Impact of withdrawal | To understand the impact of withdrawal from gene therapy on individuals and their ongoing attitude to their haemophilia care | Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach |
| Why not interested | To understand why some patients and families opt not to participate in gene therapy trials as a treatment option | Each participant & family member will take part in a 1 hour semi structured qualitative interview where his experiences of his condition, previous treatment & experience of gene therapy will be discussed & analysed using a grounded theory approach |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |