Duchenne Muscular DystrophyNCT03335384
Relationship Between PFTs and Pdi in DMD
A study testing Esophageal Balloon, Gastric Balloon and Nasal Pressure Transducer in people with Duchenne Muscular Dystrophy, run by University of Minnesota.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 to 25 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Esophageal Balloon, Gastric Balloon and Nasal Pressure Transducer
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 May 2018to May 2018
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 25 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Clinical diagnosis of Duchenne muscular dystrophy
Who cannot join
- Inability to follow verbal instructions
About this study
Official title: The Relationship Between Pulmonary Function Measures and Transdiaphragmatic Measures in Duchenne Muscular Dystrophy Subjects
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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Esophageal Balloon, Gastric Balloon, Nasal Pressure Transducer
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Esophageal BalloonDiagnostic test
Small flexible catheter with deflated balloon at distal end to be inserted into the esophagus through the nose
Gastric BalloonDiagnostic test
Small flexible catheter with deflated balloon at distal end to be inserted into the stomach through the nose
Nasal Pressure TransducerDiagnostic test
Pressure transducer inserted into nasal plug
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Diagnostic Test: Nasal Pressure Transducer |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of Pdi
For all subjects Pdi (transdiaphragmatic pressure measures) will be assessed with gastric and esophageal balloons
Measured: 1 year
Secondary outcomes (8)
Evaluation of SNIP
For all subjects, SNIP (sniff nasal inspiratory pressures) will be assessed with a nasal pressure transducer
Measured: 1 year
Evaluation of FVC
For all subjects, FVC (forced vital capacity) will be assessed with spirometry
Measured: 1 year
Evaluation of FEV1
For all subjects, FEV1 (forced expiratory volume in 1 second) will be assessed with spirometry
Measured: 1 year
Evaluation of FEFmax
For all subjects, FEFmax (maximal forced expiratory flow) will be assessed with spirometry
Measured: 1 year
Evaluation of FEF25-75
For all subjects, FEF25-75 (the average forced expiratory flow during the mid (25 - 75%) portion of the FVC) will be assessed with spirometry
Measured: 1 year
Evaluation of FEF50
For all subjects, FEF50 (forced expiratory flow at 50% of FVC) will be assessed with spirometry
Measured: 1 year
Evaluation of MIP
For all subjects, MIP (maximal inspiratory pressure) will be assessed with spirometry
Measured: 1 year
Evaluation of MEP
For all subjects, MEP (maximal expiratory pressure) will be assessed with spirometry
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2017
- Study start1 May 2018
- Primary completionMay 2018 (estimated)
- Study completionMay 2018 (estimated)
Why it stopped: Study not approved
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Cross-sectional
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- Fairview Health Services
- Responsible party
- Sponsor
- Investigators
- Eric M Snyder, PhD, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03335384
- Sponsor's protocol ID
- STUDY00001062
- Status last verified
- May 2018
- First submitted
- 12 Oct 2017
- Last updated
- 14 May 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Relationship Between Pulmonary Function Measures and Transdiaphragmatic Measures in Duchenne Muscular Dystrophy Subjects
- ClinicalTrials.gov ID
- NCT03335384
- Acronym / sponsor ID
- STUDY00001062
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study not approved
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Collaborators
- Fairview Health Services
- Study start
- 1 May 2018 (actual)
- Primary completion
- May 2018 (estimated)
- Study completion
- May 2018 (estimated)
- First posted
- 7 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Health services research
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Cross-sectional
| Arm | Type | Receives |
|---|---|---|
| Measurement of Pdi | Experimental | Diagnostic Test: Esophageal Balloon, Diagnostic Test: Gastric BalloonTwo 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 | Experimental | Diagnostic Test: Nasal Pressure TransducerWhile 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. |
Who may qualify
- Ages
- 6 to 25 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Clinical diagnosis of Duchenne muscular dystrophy
Key exclusion criteria (1 listed)
- Inability to follow verbal instructions
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Esophageal BalloonSmall flexible catheter with deflated balloon at distal end to be inserted into the esophagus through the nose | Diagnostic test | Measurement of Pdi |
| Gastric BalloonSmall flexible catheter with deflated balloon at distal end to be inserted into the stomach through the nose | Diagnostic test | Measurement of Pdi |
| Nasal Pressure TransducerPressure transducer inserted into nasal plug | Diagnostic test | Measurement of SNIPs |
Primary outcome
Evaluation of Pdi
Time frame: 1 year
For all subjects Pdi (transdiaphragmatic pressure measures) will be assessed with gastric and esophageal balloons
8 secondary outcomes
Evaluation of SNIP
Time frame: 1 year
Evaluation of FVC
Time frame: 1 year
Evaluation of FEV1
Time frame: 1 year
Evaluation of FEFmax
Time frame: 1 year
Evaluation of FEF25-75
Time frame: 1 year
Evaluation of FEF50
Time frame: 1 year
Evaluation of MIP
Time frame: 1 year
Evaluation of MEP
Time frame: 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Eric M Snyder, PhD principal investigator
University of Minnesota
Questions about this trial
What is this clinical trial studying?
A study testing Esophageal Balloon, Gastric Balloon and Nasal Pressure Transducer in people with Duchenne Muscular Dystrophy, run by University of Minnesota.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 14 May 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of Pdi (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2018.