Relationship Between PFTs and Pdi in DMD

Not Applicable

Withdrawn

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Diagnostic Test: Nasal Pressure Transducer; Diagnostic Test: Esophageal Balloon; Diagnostic Test: Gastric Balloon

• Registration Number: NCT03335384
• Lead Sponsor: University of Minnesota

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.

Study Timeline

• Study First Submitted: 2017-10-12
• Study First Submitted QC: 2017-11-02
• Study First Posted: 2017-11-07
• Status Verified: 2018-05
• Study Start: 2018-05-01
• Primary Completion: 2018-05
• Study Completion: 2018-05
• Last Update Submitted: 2018-05-08
• Last Update Posted: 2018-05-14

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: Male
• Target Recruitment: Not specified

Inclusion Criteria

• Clinical diagnosis of Duchenne muscular dystrophy

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Exclusion Criteria

• Inability to follow verbal instructions

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Measurement of Pdi	Gastric Balloon	Two small balloons, which are attached to small, flexible tubes, will be put into the esophagus (food tube) and stomach through the nose. Each balloon is about 2 inches long (deflated) and about the width of a pencil tip. A gastric balloon will be inserted into subject's stomach while an esophageal balloon will be inserted into the subject's esophagus. 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.
Measurement of SNIPs	Nasal Pressure Transducer	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 nasal pressure transducer to measure airway pressure during maximal inspiration. 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.
Measurement of Pdi	Esophageal Balloon	Two small balloons, which are attached to small, flexible tubes, will be put into the esophagus (food tube) and stomach through the nose. Each balloon is about 2 inches long (deflated) and about the width of a pencil tip. A gastric balloon will be inserted into subject's stomach while an esophageal balloon will be inserted into the subject's esophagus. 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.

Primary Outcome Measures

Name	Time	Method
Evaluation of Pdi	1 year	For all subjects Pdi (transdiaphragmatic pressure measures) will be assessed with gastric and esophageal balloons

Secondary Outcome Measures

Name	Time	Method
Evaluation of SNIP	1 year	For all subjects, SNIP (sniff nasal inspiratory pressures) will be assessed with a nasal pressure transducer
Evaluation of MEP	1 year	For all subjects, MEP (maximal expiratory pressure) will be assessed with spirometry
Evaluation of FEF50	1 year	For all subjects, FEF50 (forced expiratory flow at 50% of FVC) will be assessed with spirometry
Evaluation of FVC	1 year	For all subjects, FVC (forced vital capacity) 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
Evaluation of MIP	1 year	For all subjects, MIP (maximal inspiratory pressure) 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