Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
| Status: | Recruiting |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 20 - 60 |
| Updated: | 12/6/2018 |
| Start Date: | April 2015 |
| End Date: | December 2019 |
| Contact: | Barbara K. Smith, PT, PhD |
| Email: | bksmith@phhp.ufl.edu |
| Phone: | 352-294-5315 |
This study intends to evaluate dynamic respiratory motor performance as a valuable measure of
pulmonary function in adults with late-onset Pompe disease. The investigators will adopt a
strategy that includes comprehensive evaluations of respiratory volume, flow, and timing
parameters during resting and loaded breathing. These evaluations will then be associated to
the standard clinical measure of maximal inspiratory pressure, the static inspiratory muscle
function, as well as magnetic resonance imaging of thoracic expansion and diaphragmatic
descent at rest and with exertion. Outcomes in participants with late-onset Pompe disease
will be contrasted to the function of age- and gender-matched control subjects. This approach
will enable the investigators to evaluate the relationship between dynamic diaphragmatic
function and respiratory motor function.
pulmonary function in adults with late-onset Pompe disease. The investigators will adopt a
strategy that includes comprehensive evaluations of respiratory volume, flow, and timing
parameters during resting and loaded breathing. These evaluations will then be associated to
the standard clinical measure of maximal inspiratory pressure, the static inspiratory muscle
function, as well as magnetic resonance imaging of thoracic expansion and diaphragmatic
descent at rest and with exertion. Outcomes in participants with late-onset Pompe disease
will be contrasted to the function of age- and gender-matched control subjects. This approach
will enable the investigators to evaluate the relationship between dynamic diaphragmatic
function and respiratory motor function.
The following tests will be completed over a two-day period: Respiratory pressure tests,
breathing test, magnetic resonance imaging and magnetic resonance spectroscopy (MRI and MRS).
breathing test, magnetic resonance imaging and magnetic resonance spectroscopy (MRI and MRS).
Inclusion Criteria:
- Confirmed diagnosis of Pompe disease OR
- Be an age and gender-matched unaffected control subject
Exclusion Criteria:
- Pre-existing obstructive lung disease or asthma
- Forced vital capacity (FVC) <30% or >80% of age/gender predicted values
- Inability to travel to the study site
- Requirement for positive pressure ventilator support when awake and upright
- Participation in longitudinal studies that may independently alter lung function
- Presence of any other chronic medical condition that, in the opinion of the
investigator, would make the subject unsuitable for the study
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