Hemophilia A With InhibitorEmiandMeNCT04723693
An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families
An observational study of Hemophilia A With Inhibitor, run by Haemnet at 1 site in United Kingdom.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 8 to 100 years, men only
- Conditions the study is for
- Hemophilia A With Inhibitor
- What is being tested?
- Qualitative Interview
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 28 Feb 2020to 31 Jan 2021
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 8 to 100 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male participants with Hameophilia A and inhibitors currently on emicizumab prophylaxis and a member of their family
Who may be able to join
- Males
- A diagnosis of Haemophilia A with inhibitors
- Using emicizumab (prescribed by treating clinicians) in usual clinical care.
Who cannot join
- No history of a Factor VIII inhibitor
- Not currently being treated with emicizumab,
- Does not speak English (for the interviews)
- Does not consent to take part.
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 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.
The 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'.
Read more
This is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice.
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 therefore offers insight into how these patients and families cope with haemophilia.
Read the detailed description
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].
What 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.
This 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.
Emicizumab 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.
We 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.
- Conditions
- Hemophilia A With Inhibitor
- Being tested
- Qualitative Interview
- Keywords
- Haemophilia, 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
A single one hour semi structured qualitative interview
| Study group | Role | What they receive |
|---|---|---|
| individuals With Haemophilia A and with inhibitors on emicizumab | Qualitative interviews Receives: Other: Qualitative Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities)
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.
Measured: 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.
Secondary outcomes (3)
Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways)
To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.
Measured: 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.
Treatment Expectations
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).
Measured: 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.
Treatment Impact
To describe the impact of the change in treatment on the extended family
Measured: 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.
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 start28 Feb 2020
- Primary completion31 Jan 2021
- Study completion31 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A With InhibitorStudies 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
- Family based
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Haemnet
- Sponsor type
- University, hospital or other
- Collaborators
- Roche Chugai
- Responsible party
- Sponsor
- Investigators
- Simon P Fletcher, MA, Principal investigator, Researcher
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04723693
- Sponsor's protocol ID
- v4 4Nov2019
- Acronym
- EmiandMe
- Status last verified
- Mar 2024
- First submitted
- 14 Jan 2021
- Last updated
- 19 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34878209
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families
- ClinicalTrials.gov ID
- NCT04723693
- Acronym / sponsor ID
- EmiandMe / v4 4Nov2019
- Conditions
- Hemophilia A With Inhibitor
- Keywords
- Haemophilia, Quality of Life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- HaemnetUniversity, hospital or other
- Collaborators
- Roche Chugai
- Study start
- 28 Feb 2020 (actual)
- Primary completion
- 31 Jan 2021 (actual)
- Study completion
- 31 Jan 2021 (actual)
- First posted
- 26 Jan 2021 (actual)
- Results
- Posted 19 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
The 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'.
This is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice.
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 therefore offers insight into how these patients and families cope with haemophilia.
Detailed description
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].
What 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.
This 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.
Emicizumab 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.
We 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.
Study design
- Observational model
- Family based
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| individuals With Haemophilia A and with inhibitors on emicizumab | – | Other: Qualitative InterviewQualitative interviews |
Who may qualify
- Ages
- 8 to 100 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male participants with Hameophilia A and inhibitors currently on emicizumab prophylaxis and a member of their family
Key inclusion criteria (3 listed)
- Males
- A diagnosis of Haemophilia A with inhibitors
- Using emicizumab (prescribed by treating clinicians) in usual clinical care.
Key exclusion criteria (4 listed)
- No history of a Factor VIII inhibitor
- Not currently being treated with emicizumab,
- Does not speak English (for the interviews)
- Does not consent to take part.
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 InterviewA single one hour semi structured qualitative interview | Other | individuals With Haemophilia A and with inhibitors on emicizumab |
Primary outcome
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities)
Time frame: 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.
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.
3 secondary outcomes
Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways)
Time frame: 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.
Treatment Expectations
Time frame: 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.
Treatment Impact
Time frame: 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 34878209Fletcher 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.
- 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, MA 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 A With Inhibitor, 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 3 inclusion and 4 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 19 Aug 2024. 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?
Yes. Results are posted in the registry (first posted 19 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities) (time frame: 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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04723693, which also lists 1 linked publication.
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 19 Aug 2024.