Marfan SyndromeNCT03567460
Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
A study testing Exercise in people with Marfan Syndrome, run by Stanford University at 1 site in United States.
Trial summary
Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise. Read more about this study
- Who is it for?
- Ages 10 to 19 years
- Conditions the study is for
- Marfan Syndrome
- What is being tested?
- Exercise
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2017to 31 Dec 2019
- Phase
- Interventional
- 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 10 to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 10-19 years of age,
- MFS by revised Ghent criteria,
- Cardiac clearance to exercise by the primary cardiologist.
Who cannot join
- Ventricular dysfunction,
- Prior history of aortic surgery.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford Children's Health
Palo Alto, California, 94306
About this study
Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.
There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.
For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health in adolescent Marfan patients.
Read the detailed description
Marfan syndrome is an inherited disorder of the connective tissue, which provides material and support for the skeleton, muscles, and blood vessels. Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.
Typically, the weakening process starts when elastin fibers in the aorta become fragmented. There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.
For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. These young patients also frequently choose sedentary lifestyles, most likely due to limitations imposed by parents as well as adolescents' own perceptions of what is safe for them and their physical capabilities. Another complicating factor is that these patients often experience difficulty coping with their diagnosis. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health and coping skills in adolescent Marfan patients.
- Conditions
- Marfan Syndrome
- Being tested
- Exercise
- Medical subject headings
- Marfan Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExerciseBehavioural
Across studies in adolescents in general population, 60 minutes of recommended daily exercise level is achieved, on average, within a total volume of 10,000-11,700 steps. The investigators will assess baseline physical activity quantitatively by using an accelerometer worn on the wrist during waking hours over a 7-day period. Each participant will be given a Garmin device to place on their wrist that will track their steps. Then patients will be asked to complete 10,000 steps daily for 6 months (acclimation over a week), which will be encouraged by daily text messages or e-mail, and weekly phone calls by the intervention team in addition to a Garmin and Facebook peer group.
| Study group | Role | What they receive |
|---|---|---|
| 10,000 Steps/day for Pediatric Marfan Patients | Experimental | Participants will be given a Garmin VivoFit and asked to take at least 10,000 steps per day. A study coordinator will reach out at least once per week to check in on progress made and help make weekly goals. Receives: Behavioral: Exercise |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients.
The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).
Measured: 6 months of intervention
Secondary outcomes (4)
To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
The secondary outcomes include aortic stiffness at the aortic root at the level of the sinuses of Valsalva and ascending aorta.
Measured: 6 months of intervention
To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
Secondary outcomes will be expression levels of TGF-β, Ang-II, MMP-2 \& -9, ROS levels.
Measured: 6 months of intervention
To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.
Secondary outcomes will be expression levels of TGF-β, MMP-2, MMP-9, and ROS.
Measured: 6 months of intervention
To determine if 6 months of regular physical activity improves coping skills in Marfan patients.
The outcome measure is the COPE inventory score.
Measured: 6 months of intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2018
- Study start1 Jan 2017
- Primary completion31 Dec 2019
- Study completion31 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Marfan SyndromeStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Midwestern University
- Responsible party
- Principal investigator, Seda Tierney, Stanford University
- Investigators
- Seda Tierney, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03567460
- Sponsor's protocol ID
- 37176
- Status last verified
- Jul 2020
- First submitted
- 3 Oct 2017
- Last updated
- 27 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Selamet Tierney ES, Chung S, Stauffer KJ, Brabender J, Collins RT 2nd, Folk R, Li W, Murthy AK, Murphy DJ, Esfandiarei M. Can 10 000 Healthy Steps a Day Slow Aortic Root Dilation in Pediatric Patients With Marfan Syndrome? J Am Heart Assoc. 2022 Dec 6;11(23):e027598. doi: 10.1161/JAHA.122.027598. Epub 2022 Dec 1.
PubMed 36453629
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
- ClinicalTrials.gov ID
- NCT03567460
- Acronym / sponsor ID
- 37176
- Conditions
- Marfan Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Midwestern University
- Study start
- 1 Jan 2017 (actual)
- Primary completion
- 31 Dec 2019 (actual)
- Study completion
- 31 Dec 2019 (actual)
- First posted
- 25 Jun 2018 (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 patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.
There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.
For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health in adolescent Marfan patients.
Detailed description
Marfan syndrome is an inherited disorder of the connective tissue, which provides material and support for the skeleton, muscles, and blood vessels. Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.
Typically, the weakening process starts when elastin fibers in the aorta become fragmented. There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.
For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. These young patients also frequently choose sedentary lifestyles, most likely due to limitations imposed by parents as well as adolescents' own perceptions of what is safe for them and their physical capabilities. Another complicating factor is that these patients often experience difficulty coping with their diagnosis. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health and coping skills in adolescent Marfan patients.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 10,000 Steps/day for Pediatric Marfan Patients | Experimental | Behavioral: ExerciseParticipants will be given a Garmin VivoFit and asked to take at least 10,000 steps per day. A study coordinator will reach out at least once per week to check in on progress made and help make weekly goals. |
Who may qualify
- Ages
- 10 to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 10-19 years of age,
- MFS by revised Ghent criteria,
- Cardiac clearance to exercise by the primary cardiologist.
Key exclusion criteria (2 listed)
- Ventricular dysfunction,
- Prior history of aortic surgery.
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 |
|---|---|---|
| ExerciseAcross studies in adolescents in general population, 60 minutes of recommended daily exercise level is achieved, on average, within a total volume of 10,000-11,700 steps. The investigators will assess baseline physical activity quantitatively by using an accelerometer worn on the wrist during waking hours over a 7-day period. Each participant will be given a Garmin device to place on their wrist that will track their steps. Then patients will be asked to complete 10,000 steps daily for 6 months (acclimation over a week), which will be encouraged by daily text messages or e-mail, and weekly phone calls by the intervention team in addition to a Garmin and Facebook peer group. | Behavioural | 10,000 Steps/day for Pediatric Marfan Patients |
Primary outcome
To determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients.
Time frame: 6 months of intervention
The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).
4 secondary outcomes
To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
Time frame: 6 months of intervention
To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.
Time frame: 6 months of intervention
To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.
Time frame: 6 months of intervention
To determine if 6 months of regular physical activity improves coping skills in Marfan patients.
Time frame: 6 months of intervention
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 27 Jul 2020.
- Derived from this study: PubMed 36453629Selamet Tierney ES, Chung S, Stauffer KJ, Brabender J, Collins RT 2nd, Folk R, Li W, Murthy AK, Murphy DJ, Esfandiarei M. Can 10 000 Healthy Steps a Day Slow Aortic Root Dilation in Pediatric Patients With Marfan Syndrome? J Am Heart Assoc. 2022 Dec 6;11(23):e027598. doi: 10.1161/JAHA.122.027598. Epub 2022 Dec 1.
- Individual participant dataSharing planned: No
Related trials for Marfan Syndrome
RecruitingObservational study
Asprosin and miRNA Expression in Young Women With Metabolic Syndrome
Metabolic Syndrome, Insulin Resistance
Enrolling by invitationObservational study
Advanced Ultrasound Signal Processing of Suspicious Breast Images
Breast Cancer, Breast Cyst
RecruitingObservational study
Pregnancy in Women With Rare Multisystemic Vascular Diseases: COGRare5 Study
Vascular Anomaly, Osler Rendu Disease
Not yet recruitingObservational study
Asprosin in Behect Patients
Behcet Disease
Not yet recruitingObservational study
Serum Asprosin, Metabolic Syndrome, and Cardiometabolic Risk in Rheumatoid Arthritis
Rheumatoid Arthritis, Metabolic Syndrome
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.
Seda Tierney, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Exercise in people with Marfan Syndrome, run by Stanford University at 1 site in United States.
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 2 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 27 Jul 2020. 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 States. 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 determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients. (time frame: 6 months of intervention). 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 NCT03567460, 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 27 Jul 2020.