N/A
N=14
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Pompe Disease
Bottom Line
View on ClinicalTrials.gov: NCT02354664 ↗Enrolled (actual)
14
Serious AEs
0.0%
Results posted
Jun 2024
Primary outcome: Primary: Inspiratory Load Compensation - Inspiratory Volume — 288.41; 554.34; 743.71; 927.57 mL
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Thoracic MRI (Other); Spirometry (Other); Inspiratory Load Compensation (Other); Maximal Inspiratory Pressure (Other); Resting Breathing Pattern (Other); Respiratory Muscle Endurance Test (Other)
- Age
- Adult, Older Adult · 20+ yrs
- Sex
- All
- Sponsor
- University of Florida
- Primary completion
- Oct 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Inspiratory Load Compensation - Inspiratory Volume |
288.41; 554.34; 743.71; 927.57 | — |
| PRIMARY Respiratory Muscle Endurance Task |
2.9; 7.7 | — |
| PRIMARY Inspiratory Load Compensation - Inspiratory Flow |
18.57; 21.62; 47.05; 42.50 | — |
| PRIMARY Inspiratory Load Compensation - Inspiratory Time |
1.76; 3.80; 1.41; 1.93 | — |
| SECONDARY Thoracic MRI |
0.85; 1.88; 0.54; 0.58; 1.93; 5.77 | — |
Summary
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.
Eligibility Criteria
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) 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
Data sourced from ClinicalTrials.gov (NCT02354664). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.