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Relationship Between PFTs and Pdi in DMD

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ClinicalTrials.gov Identifier: NCT03335384
Recruitment Status : Withdrawn (Study not approved)
First Posted : November 7, 2017
Last Update Posted : May 14, 2018
Sponsor:
Collaborator:
Fairview Health Services
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Diagnostic Test: Esophageal Balloon Diagnostic Test: Gastric Balloon Diagnostic Test: Nasal Pressure Transducer Not Applicable

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Cross-sectional
Masking: None (Open Label)
Primary Purpose: Health Services Research
Official Title: The Relationship Between Pulmonary Function Measures and Transdiaphragmatic Measures in Duchenne Muscular Dystrophy Subjects
Actual Study Start Date : May 1, 2018
Estimated Primary Completion Date : May 2018
Estimated Study Completion Date : May 2018


Arm Intervention/treatment
Experimental: Measurement of Pdi
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.
Diagnostic Test: Esophageal Balloon
Small flexible catheter with deflated balloon at distal end to be inserted into the esophagus through the nose

Diagnostic Test: Gastric Balloon
Small flexible catheter with deflated balloon at distal end to be inserted into the stomach through the nose

Experimental: Measurement of SNIPs
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.
Diagnostic Test: Nasal Pressure Transducer
Pressure transducer inserted into nasal plug




Primary Outcome Measures :
  1. Evaluation of Pdi [ Time Frame: 1 year ]
    For all subjects Pdi (transdiaphragmatic pressure measures) will be assessed with gastric and esophageal balloons


Secondary Outcome Measures :
  1. Evaluation of SNIP [ Time Frame: 1 year ]
    For all subjects, SNIP (sniff nasal inspiratory pressures) will be assessed with a nasal pressure transducer

  2. Evaluation of FVC [ Time Frame: 1 year ]
    For all subjects, FVC (forced vital capacity) will be assessed with spirometry

  3. Evaluation of FEV1 [ Time Frame: 1 year ]
    For all subjects, FEV1 (forced expiratory volume in 1 second) will be assessed with spirometry

  4. Evaluation of FEFmax [ Time Frame: 1 year ]
    For all subjects, FEFmax (maximal forced expiratory flow) will be assessed with spirometry

  5. Evaluation of FEF25-75 [ Time Frame: 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

  6. Evaluation of FEF50 [ Time Frame: 1 year ]
    For all subjects, FEF50 (forced expiratory flow at 50% of FVC) will be assessed with spirometry

  7. Evaluation of MIP [ Time Frame: 1 year ]
    For all subjects, MIP (maximal inspiratory pressure) will be assessed with spirometry

  8. Evaluation of MEP [ Time Frame: 1 year ]
    For all subjects, MEP (maximal expiratory pressure) will be assessed with spirometry



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Ages Eligible for Study:   6 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of Duchenne muscular dystrophy

Exclusion Criteria:

  • Inability to follow verbal instructions

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03335384


Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Fairview Health Services
Investigators
Principal Investigator: Eric M Snyder, PhD University of Minnesota - Clinical and Translational Science Institute

Responsible Party: University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier: NCT03335384     History of Changes
Other Study ID Numbers: STUDY00001062
First Posted: November 7, 2017    Key Record Dates
Last Update Posted: May 14, 2018
Last Verified: May 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked