Marfan SyndromeNCT04774172
Mortality and Morbidity Outcomes in Marfans
An observational study of Marfan Syndrome and Marfan Syndrome Cardiovascular Manifestations, run by Barts & The London NHS Trust at 1 site in United Kingdom.
Trial summary
Marfan Syndrome (MFS) is a genetic disease affecting the eyes, skeleton, heart and arteries. Despite MFS affecting multiple organ systems, cardiovascular manifestations are the most serious and life threatening. Read more about this study
- Who is it for?
- Ages 18 to 120 years
- Conditions the study is for
- Marfan Syndrome, Marfan Syndrome Cardiovascular Manifestations
- How many people
- 6,700 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Dec 2023to 30 Jun 2024
- 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 18 to 120 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study will include all eligible patients who are identified as MFS using the ICD-10 code Q874 through the National Health Service Digital (NHSD) from January 2010 to December 2019.
Who may be able to join
- >/= 18 years old at the time of surgery
- Identified MFS in the HES database using the ICD diagnosis code Q874 between January 2010- December 2019
- Had an aorto-vascular surgery in England and Wales, as identified in the NICOR Adult Cardiac Surgery database
Who cannot join
- < 18 years old at the time of surgery
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Mortality and Morbidity Outcomes After Aortovascular Surgery in Patients With Marfan Syndrome: A UK Experience
Marfan Syndrome (MFS) is a genetic disease affecting the eyes, skeleton, heart and arteries. Despite MFS affecting multiple organ systems, cardiovascular manifestations are the most serious and life threatening. Approximately 80% of adult MFS patients will have a dilated aortic root by age 40 years with aortic aneurysm and dissection the leading causes of morbidity and mortality. Improvement in diagnostics and medical and surgical interventions have increased life expectancy. However, the natural history and the influence of medical or surgical interventions in the UK population are not fully described. Further, the incidence of aortovascular surgery in this patient group is unknown as MFS is not routinely documented in the National Institute of Cardiovascular Outcome Research (NICOR) national cardiac surgery dataset and therefore, there is currently no mechanism for exploring the aortovascular outcomes for this patient group.
The investigators aim to undertake a 10-year secondary analysis of linked national data (National Institute of Cardiovascular Outcome Research (NICOR), Office of National Statistics (ONS), Hospital Episode Statistics (HES)) to identify the UK incidence and outcome of aorto-vascular surgery in patients with Marfan syndrome (MFS). This includes associated hospital length of stay, mortality and morbidity rates.
Understanding mortality alongside morbidity will allow the investigators to study further the burdens that the aortovascular manifestations may place on MFS population as well as to continuously evaluate the efficacy of either the health care system or an implemented intervention in place. Further, these metrics will be useful for the stakeholders to effectively prioritise which complications to tackle and to allocate resources toward as well as proactively manage the potential onset of a health event
Read the detailed description
Marfan Syndrome (MFS) is a genetic disease affecting the eyes, skeleton, heart and arteries. Despite MFS affecting multiple organ systems, cardiovascular manifestations are the most serious and life threatening. Approximately 80% of adult MFS patients will have a dilated aortic root by age 40 years with aortic aneurysm and dissection the leading causes of morbidity and mortality. Improvement in diagnostics and medical and surgical interventions have increased life expectancy. However, the natural history and the influence of medical or surgical interventions in the UK population are not fully described. Further, the incidence of aortovascular surgery in this patient group is unknown as MFS is not routinely documented in the National Institute of Cardiovascular Outcome Research (NICOR) national cardiac surgery dataset and therefore, there is currently no mechanism for exploring the aortovascular outcomes for this patient group.
The investigators aim to undertake a 10-year secondary analysis of linked national data (National Institute of Cardiovascular Outcome Research (NICOR), Office of National Statistics (ONS), Hospital Episode Statistics (HES)) to identify the UK incidence and outcome of aorto-vascular surgery in patients with Marfan syndrome (MFS). This includes associated hospital length of stay, mortality and morbidity rates.
Understanding mortality alongside morbidity will allow the investigators to study further the burdens that the aortovascular manifestations may place on MFS population as well as to continuously evaluate the efficacy of either the health care system or an implemented intervention in place. Further, these metrics will be useful for the stakeholders to effectively prioritise which complications to tackle and to allocate resources toward as well as proactively manage the potential onset of a health event.
- Medical subject headings
- Marfan Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Mortality mortality rate 1-year after surgery. Mortality rate 1 year after aorto-vascular surgery
mortality rate 1-year after surgery
Measured: 1 year after surgery
Secondary outcomes (5)
Morbidity outcomes
(i) Underlying cause of death and multiple causes of death
Measured: within 1 year after surgery
Morbidity outcomes
Date of each Finished Consultant Episode
Measured: within 1 year after surgery
Morbidity outcomes
Primary and secondary diagnostic codes
Measured: within 1 year after surgery
Morbidity outcomes
Primary procedural codes
Measured: within 1 year after surgery
Morbidity outcomes
Hospital episode length
Measured: within 1 year after surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2021
- Study start1 Dec 2023
- Primary completion30 Jun 2024
- Study completion30 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Marfan SyndromeStudies 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
- Retrospective
- Enrolment
- 6,700 participants (actual)
Sponsor and investigators
- Lead sponsor
- Barts & The London NHS Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Liverpool Heart and Chest Hospital NHS Foundation Trust, Aortic Dissection Awareness UK & Ireland.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04774172
- Sponsor's protocol ID
- 294022
- Status last verified
- Aug 2024
- First submitted
- 18 Feb 2021
- Last updated
- 23 Aug 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mortality and Morbidity Outcomes After Aortovascular Surgery in Patients With Marfan Syndrome: A UK Experience
- ClinicalTrials.gov ID
- NCT04774172
- Acronym / sponsor ID
- 294022
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6,700 participants (actual)
- Lead sponsor
- Barts & The London NHS TrustUniversity, hospital or other
- Collaborators
- Liverpool Heart and Chest Hospital NHS Foundation Trust, Aortic Dissection Awareness UK & Ireland.
- Study start
- 1 Dec 2023 (actual)
- Primary completion
- 30 Jun 2024 (actual)
- Study completion
- 30 Jun 2024 (actual)
- First posted
- 1 Mar 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.
Marfan Syndrome (MFS) is a genetic disease affecting the eyes, skeleton, heart and arteries. Despite MFS affecting multiple organ systems, cardiovascular manifestations are the most serious and life threatening. Approximately 80% of adult MFS patients will have a dilated aortic root by age 40 years with aortic aneurysm and dissection the leading causes of morbidity and mortality. Improvement in diagnostics and medical and surgical interventions have increased life expectancy. However, the natural history and the influence of medical or surgical interventions in the UK population are not fully described. Further, the incidence of aortovascular surgery in this patient group is unknown as MFS is not routinely documented in the National Institute of Cardiovascular Outcome Research (NICOR) national cardiac surgery dataset and therefore, there is currently no mechanism for exploring the aortovascular outcomes for this patient group.
The investigators aim to undertake a 10-year secondary analysis of linked national data (National Institute of Cardiovascular Outcome Research (NICOR), Office of National Statistics (ONS), Hospital Episode Statistics (HES)) to identify the UK incidence and outcome of aorto-vascular surgery in patients with Marfan syndrome (MFS). This includes associated hospital length of stay, mortality and morbidity rates.
Understanding mortality alongside morbidity will allow the investigators to study further the burdens that the aortovascular manifestations may place on MFS population as well as to continuously evaluate the efficacy of either the health care system or an implemented intervention in place. Further, these metrics will be useful for the stakeholders to effectively prioritise which complications to tackle and to allocate resources toward as well as proactively manage the potential onset of a health event
Detailed description
Marfan Syndrome (MFS) is a genetic disease affecting the eyes, skeleton, heart and arteries. Despite MFS affecting multiple organ systems, cardiovascular manifestations are the most serious and life threatening. Approximately 80% of adult MFS patients will have a dilated aortic root by age 40 years with aortic aneurysm and dissection the leading causes of morbidity and mortality. Improvement in diagnostics and medical and surgical interventions have increased life expectancy. However, the natural history and the influence of medical or surgical interventions in the UK population are not fully described. Further, the incidence of aortovascular surgery in this patient group is unknown as MFS is not routinely documented in the National Institute of Cardiovascular Outcome Research (NICOR) national cardiac surgery dataset and therefore, there is currently no mechanism for exploring the aortovascular outcomes for this patient group.
The investigators aim to undertake a 10-year secondary analysis of linked national data (National Institute of Cardiovascular Outcome Research (NICOR), Office of National Statistics (ONS), Hospital Episode Statistics (HES)) to identify the UK incidence and outcome of aorto-vascular surgery in patients with Marfan syndrome (MFS). This includes associated hospital length of stay, mortality and morbidity rates.
Understanding mortality alongside morbidity will allow the investigators to study further the burdens that the aortovascular manifestations may place on MFS population as well as to continuously evaluate the efficacy of either the health care system or an implemented intervention in place. Further, these metrics will be useful for the stakeholders to effectively prioritise which complications to tackle and to allocate resources toward as well as proactively manage the potential onset of a health event.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study will include all eligible patients who are identified as MFS using the ICD-10 code Q874 through the National Health Service Digital (NHSD) from January 2010 to December 2019.
Key inclusion criteria (3 listed)
- >/= 18 years old at the time of surgery
- Identified MFS in the HES database using the ICD diagnosis code Q874 between January 2010- December 2019
- Had an aorto-vascular surgery in England and Wales, as identified in the NICOR Adult Cardiac Surgery database
Key exclusion criteria (1 listed)
- < 18 years old at the time of surgery
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Mortality mortality rate 1-year after surgery. Mortality rate 1 year after aorto-vascular surgery
Time frame: 1 year after surgery
mortality rate 1-year after surgery
5 secondary outcomes
Morbidity outcomes
Time frame: within 1 year after surgery
Morbidity outcomes
Time frame: within 1 year after surgery
Morbidity outcomes
Time frame: within 1 year after surgery
Morbidity outcomes
Time frame: within 1 year after surgery
Morbidity outcomes
Time frame: within 1 year after surgery
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 23 Aug 2024.
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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.
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 Marfan Syndrome and Marfan Syndrome Cardiovascular Manifestations, run by Barts & The London NHS Trust 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 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 23 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?
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?
Mortality mortality rate 1-year after surgery. Mortality rate 1 year after aorto-vascular surgery (time frame: 1 year after surgery). 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 23 Aug 2024.