Relationship Between PFTs and Pdi in DMD

NCT03335384 | Withdrawn DMC FDA Device

• 1 other identifier: STUDY00001062
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Evaluation of Pdi

  For all subjects Pdi (transdiaphragmatic pressure measures) will be assessed with gastric and esophageal balloons

  1 year

Secondary Outcomes (8)

• Evaluation of SNIP

  1 year

• Evaluation of FVC

  1 year

• Evaluation of FEV1

  1 year

• Evaluation of FEFmax

  1 year

• Evaluation of FEF25-75

  1 year

Study Arms (2)

Measurement of Pdi

EXPERIMENTAL

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; Diagnostic Test: Gastric Balloon

Measurement of SNIPs

EXPERIMENTAL

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

Interventions

Esophageal Balloon DIAGNOSTIC_TEST

Small flexible catheter with deflated balloon at distal end to be inserted into the esophagus through the nose

Measurement of Pdi

Gastric Balloon DIAGNOSTIC_TEST

Small flexible catheter with deflated balloon at distal end to be inserted into the stomach through the nose

Measurement of Pdi

Nasal Pressure Transducer DIAGNOSTIC_TEST

Pressure transducer inserted into nasal plug

Measurement of SNIPs

Eligibility Criteria

• Age: 6 Years - 25 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Clinical diagnosis of Duchenne muscular dystrophy

You may not qualify if:

• Inability to follow verbal instructions

Sponsors & Collaborators

• University of Minnesota: lead
• Fairview Health Services: collaborator

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Eric M Snyder, PhD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Cross-sectional

Study Record Dates

• First Submitted: October 12, 2017
• First Posted: November 7, 2017
• Study Start: May 1, 2018
• Primary Completion: May 1, 2018
• Study Completion: May 1, 2018
• Last Updated: May 14, 2018

Record last verified: 2018-05