Actigraphy in Pediatric Pulmonary Hypertension
Condition: Pediatric Pulmonary Hypertension
MeSH terms: Hypertension, Pulmonary; Hypertension
Study type: Observational

Summary: 
      Physical activity is an important factor in understanding how diseases can affect a child.
      Decreases in physical activity are sometimes the first thing that happens before a child is
      diagnosed with a disease. When a child sees their doctor, that visit reflects a single point
      in time. It does not capture how a child feels during the days in between visits to their
      doctor. This study plans to use special monitors called actigraphs to collect information
      about physical activity in children with a type of disease called pulmonary hypertension.
      This study will measure how active these children are and compare their activity to clinical
      information and to actigraphy measurements in children without pulmonary hypertension.
    

Detailed description: 
      Pediatric pulmonary arterial hypertension (PAH) (roughly 1-5 cases per 1 million children) is
      a severe disorder with high mortality and morbidity, but limited treatment options. Pediatric
      PAH is defined the same as that in adults, which is the presence of abnormally high pulmonary
      artery pressure. In comparison with adults, pediatric PAH is likely a more severe disorder
      with higher mortality and morbidity without treatment. Except for INOmax for persistent
      pulmonary hypertension of the newborn, no drug is currently approved in the US to treat
      pediatric PAH patients 1-17 years of age. Drug development to treat pediatric PAH is an unmet
      public health need for children and a priority for the Food and Drug Administration (FDA).
      However, studies that address the safety and efficacy of PAH therapies are rare, in part due
      to the lack of suitable clinical endpoints and quantitative and qualitative measures of
      disease severity.

      A major limitation towards enhancing outcomes of children with PAH is the lack of pediatric
      efficacy endpoints or surrogate measures that are capable of reproducibly and reliably
      reflecting changes in pulmonary arterial pressure in response to a therapeutic intervention
      that is being assessed in a pediatric clinical trial. Children are often too young and
      developmentally unable to perform standard cardiopulmonary exercise testing and the use of
      several metrics are not as accurate for reflecting clinical status in children as in adults,
      such as 6 minute walking distance. Additionally, the use of cardiac catheterization to
      directly measure pulmonary vascular resistance is invasive, requires anesthesia, and has
      additional risks for complications.

      Therefore, to address this critical and unmet medical need in children with PAH, we propose
      to begin develop a novel, developmentally-appropriate non-invasive endpoint in children
      through the use of actigraphy. Actigraphy is a mobile device that directly, reproducibly and
      non-invasively measures physical activity, which can be readily assessed in the ambulatory
      setting, and may provide a novel, simple and inexpensive approach. Impaired exercise
      tolerance is a prominent feature of PAH and contributes significantly to reduced quality of
      life. Assessing exercise capacity is an integral part of the clinical evaluation of PAH in
      adults and the use of the 6 minute walk distance (6MWD) is the most common primary endpoint
      in adult PAH clinical trials. Importantly, the 6MWD test and other existing exercise
      performance tests that are readily applied in adult studies are not reliable and applicable
      for young children and infants. We propose that actigraphy may provide a novel endpoint for
      assessing drug efficacy in children if proven to be strongly predictive and reflect clinical
      course and outcomes of children with PAH. If successful, this early study has great potential
      for having a significant regulatory impact that will advance the public health mission, as
      actigraphy could possibly become an endpoint that would be accepted in pediatrics as a
      regulatory standard for PAH clinical trials.

      The purpose of the current study is to describe the use of actigraphy in children with PAH
      and to determine if correlations exist between actigraphy data and clinical data in children
      with PAH.
    

Intervention name(s): No interventions / observations only
Intervention type(s): Other
Intervention description(s): This is an observational study only
Other intervention name(s): There are no interventions as part of this study
Observational model: Case-Control
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        We will include children with pulmonary hypertension and children without pulmonary
        hypertension into this study. All enrolled children must be between the ages of 8-14 years
        at the time of enrollment.