HemophiliaExigencyNCT04723680
An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
An observational study of Hemophilia, run by Haemnet at 1 site in United Kingdom.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 16 to 100 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- Qualitative interview
- How many people
- 69 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 13 Aug 2020to 28 Feb 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 100 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: People with haemophilia and a member of their family
Who may be able to join
- People with haemophilia A or B who consented to and have undergone gene therapy in the early dose-finding studies and a member of their family
- People with haemophilia A or B who consented to a gene therapy trial following the results of the early studies and a member of their family
- People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family
- People with haemophilia A or B who are definitely not interested in or unaware of gene therapy and a member of their family
- People with haemophilia A or B who are interested in but have not been offered gene therapy
- Those who have given written consent to be in the study
- All participants will be ≥16 years.
Who cannot join
- Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, OX3 7LE
About this study
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.
Read the detailed description
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.
- Conditions
- Hemophilia
- Being tested
- Qualitative interview
- Keywords
- Haemophilia, Gene Therapy, Quality of Life
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Qualitative interviewOther
Qualitative semi structured interview
| Study group | Role | What they receive |
|---|---|---|
| Early dose finding cohort | People with Haemophilia who took part in the early dose finding studies Receives: Other: Qualitative interview | |
| Subsequent studies group cohort | People with haemophilia who took part in subsequent gene therapy studies Receives: Other: Qualitative interview | |
| Withdrawn/ineligible Cohort | People with haemophilia who were withdrawn or proved ineligible Receives: Other: Qualitative interview | |
| Not interested cohort | People with haemophilia who are definitely not interested in gene therapy Receives: Other: Qualitative interview | |
| Not offered cohort | People with haemophilia who are interested in gene therapy but have not been the opportunity to take part Receives: Other: Qualitative interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Secondary outcomes (5)
Lived experience
To understand the lived experience of people who have undergone gene therapy
Measured: 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 gene therapy
To understand the as yet "unseen" impact of gene therapy on the extended family
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2021
- Study start13 Aug 2020
- Primary completion28 Feb 2023
- Study completion28 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Haemnet
- Sponsor type
- University, hospital or other
- Collaborators
- UniQure Biopharma B.V.
- Responsible party
- Sponsor
- Investigators
- Simon P Fletcher, Principal investigator, Researcher
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04723680
- Sponsor's protocol ID
- v3Nov2020
- Acronym
- Exigency
- Status last verified
- Feb 2023
- First submitted
- 15 Jan 2021
- Last updated
- 1 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families
- ClinicalTrials.gov ID
- NCT04723680
- Acronym / sponsor ID
- Exigency / v3Nov2020
- Conditions
- Hemophilia
- Keywords
- Haemophilia, Gene Therapy, Quality of Life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- HaemnetUniversity, hospital or other
- Collaborators
- UniQure Biopharma B.V.
- Study start
- 13 Aug 2020 (actual)
- Primary completion
- 28 Feb 2023 (actual)
- Study completion
- 28 Feb 2023 (actual)
- First posted
- 26 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Early dose finding cohort | – | Other: Qualitative interviewPeople with Haemophilia who took part in the early dose finding studies |
| Subsequent studies group cohort | – | Other: Qualitative interviewPeople with haemophilia who took part in subsequent gene therapy studies |
| Withdrawn/ineligible Cohort | – | Other: Qualitative interviewPeople with haemophilia who were withdrawn or proved ineligible |
| Not interested cohort | – | Other: Qualitative interviewPeople with haemophilia who are definitely not interested in gene therapy |
| Not offered cohort | – | Other: Qualitative interviewPeople with haemophilia who are interested in gene therapy but have not been the opportunity to take part |
Who may qualify
- Ages
- 16 to 100 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- People with haemophilia and a member of their family
Key inclusion criteria (7 listed)
- People with haemophilia A or B who consented to and have undergone gene therapy in the early dose-finding studies and a member of their family
- People with haemophilia A or B who consented to a gene therapy trial following the results of the early studies and a member of their family
- People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family
- People with haemophilia A or B who are definitely not interested in or unaware of gene therapy and a member of their family
- People with haemophilia A or B who are interested in but have not been offered gene therapy
Key exclusion criteria (1 listed)
- Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Qualitative interviewQualitative semi structured interview | Other | Early dose finding cohort, Not interested cohort, Not offered cohort, Subsequent studies group cohort, Withdrawn/ineligible Cohort |
Primary outcome
patient expectations
Time frame: 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
To explore the expectations that patients, and families in the UK have of gene therapy and its position in contemporary and future haemophilia management
5 secondary outcomes
Lived experience
Time frame: 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 gene therapy
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Simon P Fletcher principal investigator
Researcher
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Haemnet at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
patient expectations (time frame: 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Mar 2023.