HemophiliaNCT05314751
Learning to Live With Non-severe Haemophilia
An observational study of Hemophilia, run by Haemnet at 1 site in United Kingdom.
Trial summary
While the burden of standard treatment may be reduced through the use of gene therapy, converting those with severe haemophilia to a mild or moderate phenotype, the long-term sequelae of previous joint bleeds and associated limitations imposed on those with severe haemophilia may not translate to lessen the biomedical… 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?
- Questionnaire and interview
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 165 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 21 Dec 2022to 29 Sep 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 A or B either mild moderate or severe living in the UK.
Who may be able to join
- Confirmed diagnosis of haemophilia A or B of any severity
Who cannot join
- Diagnosis of any other bleeding disorder
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, OX3 9DU
About this study
While the burden of standard treatment may be reduced through the use of gene therapy, converting those with severe haemophilia to a mild or moderate phenotype, the long-term sequelae of previous joint bleeds and associated limitations imposed on those with severe haemophilia may not translate to lessen the biomedical burden of living with a history of severe haemophilia.
We wish to explore these issues further in the Learning to Live study. The study will also seek to identify the ongoing support needs of those who transition to a milder bleeding phenotype.
Read the detailed description
Haemophilia is a rare congenital disorder caused by an inherited genetic defect which affects approximately one in every 5,000 males. Haemophilia A (factor VIII [FVIII] deficiency) occurs in 85% of cases with haemophilia B (factor IX [FIX] deficiency) making up the remainder of affected people. Three severities are reported: "severe" (factor activity is less than 1%), "moderate" (factor activity is 1-5% and "mild" (factor activity is 6-25%).
The aim of haemophilia treatment is to reduce the frequency of bleeds, and consequently morbidity and joint damage, in order to prevent future disability. Historically treatment has been replacement of the missing clotting factor when a bleed occurs (on-demand treatment) or regularly and intermittently (prophylactic treatment). With this in developed countries, life expectancy of persons with haemophilia, is expected to be close to normal. Treatment is burdensome for affected individuals and caregivers with treatment complications, (presence of inhibitors, pain, and arthropathy), psychological (stress and coping, anxiety and depression, stigmatisation and discrimination) and economic aspects.
Whilst those with mild haemophilia usually only experience bleeding with trauma or surgery and are less engaged with haemophilia care, the impact of living with mild haemophilia on quality of life is becoming more recognised and further research within this cohort of people is required.
The present day focus of treatment is on normalising individual's lives and reducing treatment burden by using innovative therapies to limit and/or eliminate bleeding episodes. This results in a cohort of individuals who are now living with a less severe phenotype of haemophilia, who may be less able to recognise and treat bleeds and for whom clinical follow up and outcomes will be considerably different to that of their predecessors.
A holistic approach to follow up is required, and should include patient relevant outcome. Living with mild haemophilia is still limiting and when bleeds occur the impact is not mild; many cannot self-infuse, do not recognise when to attend hospital and suffer pain and mobility issues.
This study, which has been co-created with a person with haemophilia, aims to explore the impact of these new treatments using mixed methods study. This includes quantitative data from patient reported outcome assessment (the PROBE validated haemophilia questionnaire) and through in depth qualitative interviews.
- Conditions
- Hemophilia
- Being tested
- Questionnaire and interview
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Questionnaire and interviewBehavioural
An online survey and an in-depth interview.
What is being measured
The primary outcome is the main question the study is designed to answer.
To Identify and understand the differences between those with genetically mild/moderate haemophilia and those with previously severe haemophilia who have received phenotype altering treatment
Survey using the validated PROBE questionnaire (using 7 point Likert scale)
Measured: 3 months
Secondary outcomes (1)
To assess the impact of phenotype altering treatments on those who receive them, and to compare this with those people with genetically mild/moderate haemophilia.
Interview
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2022
- Study start21 Dec 2022
- Primary completion29 Sep 2023
- Study completion29 Sep 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
- 165 participants (actual)
Sponsor and investigators
- Lead sponsor
- Haemnet
- Sponsor type
- University, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05314751
- Sponsor's protocol ID
- L2L
- Status last verified
- Mar 2024
- First submitted
- 29 Mar 2022
- Last updated
- 13 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Participant data will not be shared beyond the study group.
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Learning to Live With Non-severe Haemophilia
- ClinicalTrials.gov ID
- NCT05314751
- Acronym / sponsor ID
- L2L
- Conditions
- Hemophilia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 165 participants (actual)
- Lead sponsor
- HaemnetUniversity, hospital or other
- Collaborators
- BioMarin Pharmaceutical
- Study start
- 21 Dec 2022 (actual)
- Primary completion
- 29 Sep 2023 (actual)
- Study completion
- 29 Sep 2023 (actual)
- First posted
- 6 Apr 2022 (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.
While the burden of standard treatment may be reduced through the use of gene therapy, converting those with severe haemophilia to a mild or moderate phenotype, the long-term sequelae of previous joint bleeds and associated limitations imposed on those with severe haemophilia may not translate to lessen the biomedical burden of living with a history of severe haemophilia.
We wish to explore these issues further in the Learning to Live study. The study will also seek to identify the ongoing support needs of those who transition to a milder bleeding phenotype.
Detailed description
Haemophilia is a rare congenital disorder caused by an inherited genetic defect which affects approximately one in every 5,000 males. Haemophilia A (factor VIII [FVIII] deficiency) occurs in 85% of cases with haemophilia B (factor IX [FIX] deficiency) making up the remainder of affected people. Three severities are reported: "severe" (factor activity is less than 1%), "moderate" (factor activity is 1-5% and "mild" (factor activity is 6-25%).
The aim of haemophilia treatment is to reduce the frequency of bleeds, and consequently morbidity and joint damage, in order to prevent future disability. Historically treatment has been replacement of the missing clotting factor when a bleed occurs (on-demand treatment) or regularly and intermittently (prophylactic treatment). With this in developed countries, life expectancy of persons with haemophilia, is expected to be close to normal. Treatment is burdensome for affected individuals and caregivers with treatment complications, (presence of inhibitors, pain, and arthropathy), psychological (stress and coping, anxiety and depression, stigmatisation and discrimination) and economic aspects.
Whilst those with mild haemophilia usually only experience bleeding with trauma or surgery and are less engaged with haemophilia care, the impact of living with mild haemophilia on quality of life is becoming more recognised and further research within this cohort of people is required.
The present day focus of treatment is on normalising individual's lives and reducing treatment burden by using innovative therapies to limit and/or eliminate bleeding episodes. This results in a cohort of individuals who are now living with a less severe phenotype of haemophilia, who may be less able to recognise and treat bleeds and for whom clinical follow up and outcomes will be considerably different to that of their predecessors.
A holistic approach to follow up is required, and should include patient relevant outcome. Living with mild haemophilia is still limiting and when bleeds occur the impact is not mild; many cannot self-infuse, do not recognise when to attend hospital and suffer pain and mobility issues.
This study, which has been co-created with a person with haemophilia, aims to explore the impact of these new treatments using mixed methods study. This includes quantitative data from patient reported outcome assessment (the PROBE validated haemophilia questionnaire) and through in depth qualitative interviews.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
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 A or B either mild moderate or severe living in the UK.
Key inclusion criteria (1 listed)
- Confirmed diagnosis of haemophilia A or B of any severity
Key exclusion criteria (1 listed)
- Diagnosis of any other bleeding disorder
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 |
|---|---|---|
| Questionnaire and interviewAn online survey and an in-depth interview. | Behavioural | – |
Primary outcome
To Identify and understand the differences between those with genetically mild/moderate haemophilia and those with previously severe haemophilia who have received phenotype altering treatment
Time frame: 3 months
Survey using the validated PROBE questionnaire (using 7 point Likert scale)
1 secondary outcome
To assess the impact of phenotype altering treatments on those who receive them, and to compare this with those people with genetically mild/moderate haemophilia.
Time frame: 3 months
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 13 Mar 2024.
- Individual participant dataSharing planned: No. Participant data will not be shared beyond the study group.
Related trials for Hemophilia
Not yet recruitingPhase 1
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A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
Hemophilia A, Healthy Adult Participants
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A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A
Haemophilia A
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
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RecruitingObservational study
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
Hemophilia A
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.
The record lists no contact. Use the official record to reach the sponsor.
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 1 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 13 Mar 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?
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?
To Identify and understand the differences between those with genetically mild/moderate haemophilia and those with previously severe haemophilia who have received phenotype altering treatment (time frame: 3 months). 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 13 Mar 2024.