Duchenne Muscular Dystrophy
Conditions
Brief summary
A cross-sectional study to explore the relationship between clinically assessed pulmonary function test (PFT) measures and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy (DMD) as well as to explore the relationship between sniff nasal inspiratory pressure (SNIP) and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy.
Detailed description
Two small balloons, which are attached to small, flexible tubes, will be put into the esophagus and stomach through your nose. Each balloon is about 2 inches long (deflated) and about the width of a pencil tip. To reduce any discomfort with this procedure, lidocaine gel or spray will be put into the subject's nose and administered to the back of the throat before the balloon. In addition, swallowing water during the procedure will help to reduce any gagging sensation and will assure that the balloon goes into the esophagus. Proper placement of the catheters will be determined using normal tidal breathing against an occluded mouthpiece with a nose clip. Once proper placement is ensured, the catheters will remain in place during your normal PFT maneuvers. Additionally, while the gastric and esophageal balloon catheters are in place, the subject will be asked to perform a maximal sniff maneuver (SNIP) while one nostril is occluded with a plug containing a distal pressure measurement catheter to measure airway pressure. The distal end of the pressure catheter will be connected to a hand held pressure meter to display peak pressure and to provide you visual feedback. This maneuver will be performed 10 times.
Interventions
DIAGNOSTIC_TESTEsophageal Balloon
Small flexible catheter with deflated balloon at distal end to be inserted into the esophagus through the nose
DIAGNOSTIC_TESTGastric Balloon
Small flexible catheter with deflated balloon at distal end to be inserted into the stomach through the nose
DIAGNOSTIC_TESTNasal Pressure Transducer
Pressure transducer inserted into nasal plug
Sponsors
Fairview Health Services
University of Minnesota
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Intervention model description
Cross-sectional
Eligibility
Sex/Gender
MALE
Age
6 Years to 25 Years
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of Duchenne muscular dystrophy
Exclusion criteria
* Inability to follow verbal instructions
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of Pdi | 1 year | For all subjects Pdi (transdiaphragmatic pressure measures) will be assessed with gastric and esophageal balloons |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of FVC | 1 year | For all subjects, FVC (forced vital capacity) will be assessed with spirometry |
| Evaluation of FEV1 | 1 year | For all subjects, FEV1 (forced expiratory volume in 1 second) will be assessed with spirometry |
| Evaluation of FEFmax | 1 year | For all subjects, FEFmax (maximal forced expiratory flow) will be assessed with spirometry |
| Evaluation of SNIP | 1 year | For all subjects, SNIP (sniff nasal inspiratory pressures) will be assessed with a nasal pressure transducer |
| Evaluation of FEF50 | 1 year | For all subjects, FEF50 (forced expiratory flow at 50% of FVC) will be assessed with spirometry |
| Evaluation of MIP | 1 year | For all subjects, MIP (maximal inspiratory pressure) will be assessed with spirometry |
| Evaluation of MEP | 1 year | For all subjects, MEP (maximal expiratory pressure) will be assessed with spirometry |
| Evaluation of FEF25-75 | 1 year | For all subjects, FEF25-75 (the average forced expiratory flow during the mid (25 - 75%) portion of the FVC) will be assessed with spirometry |
Outcome results
None listed