Marfan SyndromeNCT03581682
Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?
A study testing Tele-Clinic Visits Using Parent-Acquired Echos in people with Marfan Syndrome, run by Stanford University at 1 site in United States.
Trial summary
Marfan syndrome (MFS), a connective tissue disorder seen in 1 in 3,000 individuals, causes progressive aortic root dilation that can result in aortic dissection and sudden death. Read more about this study
- Who is it for?
- Ages 5 to 19 years
- Conditions the study is for
- Marfan Syndrome
- What is being tested?
- Tele-Clinic Visits Using Parent-Acquired Echos
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jul 2018to 1 Aug 2020
- 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 5 to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 5-19 years of age (patient)
- seen in at least 2 prior clinic visits
- Marfan syndrome by revised Ghent criteria
- presence of parent at home
Who cannot join
- prior aortic surgery
- known cardiomyopathy
- known arrhythmia
- aortic root > 4.5 cm in prior clinic visit
- pregnancy
Trial locations and contacts
1 site in 1 country.
United States1 site
Lucile Packard Children's Hospital
Palo Alto, California, 94304
About this study
Marfan syndrome (MFS), a connective tissue disorder seen in 1 in 3,000 individuals, causes progressive aortic root dilation that can result in aortic dissection and sudden death. Clinical care focuses on monitoring the aortic root by serial echocardiography (echo) to guide medical treatment and elective aortic root surgery in a specialized clinic every 6-12 months. This monitoring protocol, coupled with surgical intervention, has doubled the median life expectancy which was previously only 32 years. However, this surveillance carries significant health care costs at >$50 million dollars/year on echos alone (at $3-4K each) in children and adolescents in the US, as well as substantial burden on families residing far from specialized centers. A clinic visit delivered to MFS patients via live-video conferencing at home (tele-visit) could shift this paradigm, if a home echo could be obtained.
Here, the investigator will train parents of Pediatric Marfan patients to take echo images using a hand held device, height, weight, blood pressure, medical history, and listen to the heart of their child. Then, the investigators will ask them to take the equipment home and collect the same data at home during a tele-clinic visit, with further instruction by the study team through secure live-video conferencing.
Read the detailed description
In the proposed intervention, every patient (n=60) will have a tele-visit and an on-site clinic visit 1 day apart. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that will be used during the tele-visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo (transferred via Internet for remote interpretation), all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other. Two independent echo readers will analyze home and clinic echos to measure reproducibility.
- Conditions
- Marfan Syndrome
- Being tested
- Tele-Clinic Visits Using Parent-Acquired Echos
- Medical subject headings
- Marfan Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tele-Clinic Visits Using Parent-Acquired EchosOther
Every patient will have a tele-visit and an on-site clinic visit 1 day apart 3-6 months after the training session. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that they will use during the tele-visit. The parents will also have an in-service on how to take weight, height, and blood pressure measurements, and how to use the digital stethoscope.
A tele-visit will be schedule a day prior to the patient's regularly scheduled clinic visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo, all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other.
| Study group | Role | What they receive |
|---|---|---|
| Tele-Visit Using Parental Home Echo | Experimental | All parents will have hands-on echo training. We will test if a tele-visit using parental home echo is clinically reliable compared to an on-site clinic visit, costs less, and improves parental sense of empowerment. Receives: Other: Tele-Clinic Visits Using Parent-Acquired Echos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent of tele-visits with a clinical visit match-score of ≥13/15 points
This scoring system was determined after surveying pediatric MFS physicians nationwide and includes these sections: Echo, vital signs, height and weight, interim medical history, cardiac auscultation, and determination of follow up time and medication dose/type.
Measured: 3-6 months
Secondary outcomes (4)
To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
The outcome will be the costs of tele-visit and clinic visit. Two cost components will be estimated and combined costs will be compared: (1) Payer cost: Actual cost to deliver a tele-visit with home echo (as it is not currently covered by insurance) vs.
Medical or private insurance reimbursement rates for clinic visit with an echo; and (2) Patient-cost: time and travel costs for parents.
Measured: 3-6 months
To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
Secondary outcome will be lost school time for the patient.
Measured: 3-6 months
To determine if this intervention increases parental sense of empowerment.
The outcome will be the change in Family Empowerment Score (a validated questionnaire to assess empowerment in parents of children with illness) from study start to study end.
Measured: 3-6 months
To determine if this intervention increases parental sense of empowerment.
Secondary outcomes will be parent/patient satisfaction survey results for televisits and clinic visits using the CAHPS survey for Children, as well as patient, parent and physician interviews.
Measured: 3-6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2018
- Study start1 Jul 2018
- Primary completion1 Aug 2020
- Study completion1 Aug 2020
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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Seda Tierney, Stanford University
- Investigators
- Seda Tierney, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03581682
- Sponsor's protocol ID
- 42159
- Status last verified
- Mar 2021
- First submitted
- 18 Jun 2018
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Chen A, Punn R, Collins RT, Chen JH, Stauffer KJ, Wang R, Alexander S, MacMillen Lechich K, Murphy DJ, Chung S, Selamet Tierney ES. Tele-Clinic Visits in Pediatric Patients with Marfan Syndrome Using Parentally Acquired Echocardiography. J Pediatr. 2021 May;232:140-146. doi: 10.1016/j.jpeds.2021.01.004. Epub 2021 Jan 13.
PubMed 33453199
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?
- ClinicalTrials.gov ID
- NCT03581682
- Acronym / sponsor ID
- 42159
- Conditions
- Marfan Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- 1 Jul 2018 (actual)
- Primary completion
- 1 Aug 2020 (actual)
- Study completion
- 1 Aug 2020 (actual)
- First posted
- 10 Jul 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 syndrome (MFS), a connective tissue disorder seen in 1 in 3,000 individuals, causes progressive aortic root dilation that can result in aortic dissection and sudden death. Clinical care focuses on monitoring the aortic root by serial echocardiography (echo) to guide medical treatment and elective aortic root surgery in a specialized clinic every 6-12 months. This monitoring protocol, coupled with surgical intervention, has doubled the median life expectancy which was previously only 32 years. However, this surveillance carries significant health care costs at >$50 million dollars/year on echos alone (at $3-4K each) in children and adolescents in the US, as well as substantial burden on families residing far from specialized centers. A clinic visit delivered to MFS patients via live-video conferencing at home (tele-visit) could shift this paradigm, if a home echo could be obtained.
Here, the investigator will train parents of Pediatric Marfan patients to take echo images using a hand held device, height, weight, blood pressure, medical history, and listen to the heart of their child. Then, the investigators will ask them to take the equipment home and collect the same data at home during a tele-clinic visit, with further instruction by the study team through secure live-video conferencing.
Detailed description
In the proposed intervention, every patient (n=60) will have a tele-visit and an on-site clinic visit 1 day apart. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that will be used during the tele-visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo (transferred via Internet for remote interpretation), all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other. Two independent echo readers will analyze home and clinic echos to measure reproducibility.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tele-Visit Using Parental Home Echo | Experimental | Other: Tele-Clinic Visits Using Parent-Acquired EchosAll parents will have hands-on echo training. We will test if a tele-visit using parental home echo is clinically reliable compared to an on-site clinic visit, costs less, and improves parental sense of empowerment. |
Who may qualify
- Ages
- 5 to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 5-19 years of age (patient)
- seen in at least 2 prior clinic visits
- Marfan syndrome by revised Ghent criteria
- presence of parent at home
Key exclusion criteria (5 listed)
- prior aortic surgery
- known cardiomyopathy
- known arrhythmia
- aortic root > 4.5 cm in prior clinic visit
- pregnancy
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 |
|---|---|---|
| Tele-Clinic Visits Using Parent-Acquired EchosEvery patient will have a tele-visit and an on-site clinic visit 1 day apart 3-6 months after the training session. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that they will use during the tele-visit. The parents will also have an in-service on how to take weight, height, and blood pressure measurements, and how to use the digital stethoscope. A tele-visit will be schedule a day prior to the patient's regularly scheduled clinic visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo, all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other. | Other | Tele-Visit Using Parental Home Echo |
Primary outcome
Percent of tele-visits with a clinical visit match-score of ≥13/15 points
Time frame: 3-6 months
This scoring system was determined after surveying pediatric MFS physicians nationwide and includes these sections: Echo, vital signs, height and weight, interim medical history, cardiac auscultation, and determination of follow up time and medication dose/type.
4 secondary outcomes
To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
Time frame: 3-6 months
To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
Time frame: 3-6 months
To determine if this intervention increases parental sense of empowerment.
Time frame: 3-6 months
To determine if this intervention increases parental sense of empowerment.
Time frame: 3-6 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 17 Mar 2021.
- Derived from this study: PubMed 33453199Chen A, Punn R, Collins RT, Chen JH, Stauffer KJ, Wang R, Alexander S, MacMillen Lechich K, Murphy DJ, Chung S, Selamet Tierney ES. Tele-Clinic Visits in Pediatric Patients with Marfan Syndrome Using Parentally Acquired Echocardiography. J Pediatr. 2021 May;232:140-146. doi: 10.1016/j.jpeds.2021.01.004. Epub 2021 Jan 13.
- Individual participant dataSharing planned: Undecided
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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.
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 Tele-Clinic Visits Using Parent-Acquired Echos 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 4 inclusion and 5 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 17 Mar 2021. 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?
Percent of tele-visits with a clinical visit match-score of ≥13/15 points (time frame: 3-6 months). 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 NCT03581682, 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 17 Mar 2021.