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Trial record 38 of 88 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

Relationship Between PFTs and Pdi in DMD

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ClinicalTrials.gov Identifier: NCT03335384
Recruitment Status : Not yet recruiting
First Posted : November 7, 2017
Last Update Posted : November 7, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
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
Duchenne Muscular Dystrophy Diagnostic Test: Esophageal Balloon Diagnostic Test: Gastric Balloon Diagnostic Test: Nasal Pressure Transducer

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 Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 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
Anticipated Study Start Date : December 2017
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : May 2019


Arms and Interventions

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


Outcome Measures

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


Eligibility Criteria

Information from the National Library of Medicine

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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
Contacts and Locations

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


Contacts
Contact: Eli F Kelley, MS 320-290-0291 kelle833@d.umn.edu
Contact: Eric M Snyder, PhD 507-254-0723 snyd018@umn.edu

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
  Study Documents (Full-Text)

Documents provided by University of Minnesota - Clinical and Translational Science Institute:
More Information

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: November 7, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

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