- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03581682
Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Contacts and Locations
Study Locations
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California
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Palo Alto, California, United States, 94304
- Lucile Packard Children's Hospital
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- prior aortic surgery
- known cardiomyopathy
- known arrhythmia
- aortic root > 4.5 cm in prior clinic visit
- pregnancy
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Tele-Visit Using Parental Home Echo
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.
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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. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
3-6 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
Time Frame: 3-6 months
|
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. |
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
|
Secondary outcome will be lost school time for the patient.
|
3-6 months
|
To determine if this intervention increases parental sense of empowerment.
Time Frame: 3-6 months
|
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.
|
3-6 months
|
To determine if this intervention increases parental sense of empowerment.
Time Frame: 3-6 months
|
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.
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3-6 months
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Seda Tierney, MD, Stanford University
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Heart Diseases
- Cardiovascular Diseases
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Connective Tissue Diseases
- Bone Diseases
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Musculoskeletal Abnormalities
- Abnormalities, Multiple
- Bone Diseases, Developmental
- Limb Deformities, Congenital
- Marfan Syndrome
- Arachnodactyly
Other Study ID Numbers
- 42159
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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