- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05442671
iTONE Trial (exercIse Training in pulmONary hypertEnsion) Exercise Program for Pediatric PH (iTONE)
Home Exercise Training in Pediatric Pulmonary Hypertension
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Pediatric pulmonary hypertension (PH) is a rare disease, affecting 2-16 per million children. Without treatment, pulmonary vascular disease leads to right ventricular cardiac failure and death. Survival has improved with pharmacologic advances, however, 5-year survival from diagnosis is still only about 75%. Even with increased survival, pediatric PH is a severe, chronic illness with physical and psychosocial burdens affecting the quality of life of children and families. Exercise intolerance is nearly universal and adversely affects quality of life. While exercise physiology is complex, skeletal muscle deficits and dysfunction are important factors associated with exercise intolerance in adults with PH. The contraction of the skeletal muscle pump helps increase pulmonary blood flow at the initiation of upright exercise in the normal circulation. Low skeletal muscle mass and muscle dysfunction may impair this mechanism in PH. Investigators previously demonstrated low skeletal muscle mass in association with worse exercise performance in children with PH and identified this as an intervention target. As children with PH engage in less moderate and vigorous physical activity compared to healthy peers, we hypothesize that increasing physical activity could increase skeletal muscle mass and lead to improved exercise performance.
Recent trials have demonstrated improved exercise performance and functional class in adults with PH undertaking prescribed exercise training. Pediatric data are limited to a single, small study in which exercise performance improved after a 16-week home exercise program. That intervention did not include skeletal muscle assessment, wearable activity monitors, or specified behavioral theory, as does the current proposal. The characteristics of the optimal PH exercise program are not clear as prior studies often failed to provide sufficient detail to enable reproducibility. Most programs have been hospital-based, some with additional at-home components. Duration has varied from 4-15 weeks. Programs have included aerobic, resistance, and respiratory exercise. Patient adherence has been inconsistently assessed. While wearable activity monitors have been used to assess activities of daily living of PH patients, they have not been employed in PH exercise programs. In youth, practical concerns of developmental differences, access to facilities or equipment, and parental time commitment must be considered, and home programs may be critical to avoiding missed school and work.
Newer technologic advances enable remote monitoring of physical activity in the home setting. Wearable accelerometer sensors with wireless transmission capabilities allow us to measure relevant endpoints from a patient's daily life. In this study, participants will undergo baseline assessment of muscle mass and strength, 6-minute walk test distance, and quality of life prior to a 16-week home exercise program, enriched by mobile health technology. Investigators will use a wrist-worn accelerometer paired with periodic text messages to measure activity, obtain daily downloads, and engage patients to promote adherence. Assessment of muscle mass and strength, 6-minute walk test distance, and quality of life will be repeated after the intervention. This real-time monitoring of adherence and feedback to the patient represent fundamental changes in PH treatment paradigms that can significantly enhance efficacy.
Study Type
Enrollment (Estimated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Catherine Avitabile, MD
- Phone Number: 215-590-4040
- Email: avitabilec@chop.edu
Study Locations
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19104
- Recruiting
- Children's Hospital of Philadelphia
-
Contact:
- Catherine Avitabile, MD
- Email: avitabilec@chop.edu
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Age 8-18 years
- PH World Health Organization (WHO) diagnostic groups 1, 2, 3, or 4 (pulmonary arterial hypertension, PH due to left heart disease, PH due to lung disease, chronic thromboembolic PH)
- WHO functional class I or II
- Ambulatory status
- Mean pulmonary to systemic arterial pressure ratio <0.75 on most recent cardiac catheterization
- Completion of a cardiopulmonary exercise (CPET) test within 3 months
- Stable PH medication regimen for 3 months prior to the intervention
- Home Wifi connection
- Mobile device in family capable of receiving text messages
Exclusion Criteria:
- WHO functional class III or IV
- Single ventricle physiology
- Moderate to severe renal disease (>stage 3)
- Severe hepatic impairment [aspartate aminotransferase (AST)/alanine transaminase (ALT) > 2x upper limit of normal]
- Current pregnancy
- Significant developmental delay/inability to comply with verbal instructions to complete the study procedures
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: Home exercise intervention
|
16-week home exercise intervention, enriched by mobile health technology
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in moderate to vigorous physical activity (MVPA)
Time Frame: Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Change in MVPA (min/day) will be measured by smartwatch accelerometer from baseline over the course of the intervention
|
Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in leg lean mass Z-score (LLMZ)
Time Frame: Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
LLMZ will be measured by densitometry.
LLMZ is a percentile score with scores -2 to 2 considered typical
|
Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Change in muscle strength
Time Frame: Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Upper extremity strength will be measured by handgrip dynamometer.
Lower extremity strength will be measured by Biodex across the knee and ankle
|
Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Change in 6-minute walk test distance
Time Frame: Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
6-minute walk distance (in meters) will be measured per standard American Thoracic Society guidelines
|
Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Change in quality of life score
Time Frame: Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Score measured by completion of PedsQL questionnaire.
Scores range from 0-100 with higher scores indicating better quality of life
|
Pre-exercise intervention and Post intervention (within 2 weeks of completion of the 16-week exercise intervention)
|
Collaborators and Investigators
Collaborators
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 20-018168
- 1K23HL150337-01A1 (U.S. NIH Grant/Contract)
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
product manufactured in and exported from the U.S.
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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