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Trial record 24 of 103 for:    Pompe Disease

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease (DRMF)

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ClinicalTrials.gov Identifier: NCT02354664
Recruitment Status : Recruiting
First Posted : February 3, 2015
Last Update Posted : December 4, 2018
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
University of Florida

Tracking Information
First Submitted Date January 21, 2015
First Posted Date February 3, 2015
Last Update Posted Date December 4, 2018
Actual Study Start Date April 2015
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 29, 2015)
  • Inspiratory load compensation [ Time Frame: Day 1 ]
    Peak inspiratory flow, inspiratory volume, and duty cycle of loaded breaths will be measured
  • Respiratory muscle endurance task [ Time Frame: Day 2 ]
    Time and load associated with endurance task failure will be measured.
  • Inspiratory load Compensation [ Time Frame: Day 2 ]
    Peak inspiratory flow, inspiratory volume, and duty cycle of loaded breaths will be measured
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02354664 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: January 29, 2015)
Thoracic MRI [ Time Frame: Day 1 ]
Chest wall and diaphragmatic motions will be measured in three planes with dynamic magnetic resonance imaging (MRI). The change in diaphragm excursion between full inspiration and full expiration will be measured in the frontal plane, and change in the area of the right and left sides of the chest cavity will be measured in the sagittal plane. Diaphragm and chest excursion will be recorded dynamically during 30-second periods of resting breathing and deep breathing.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Official Title Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Brief Summary This study intends to evaluate dynamic respiratory motor performance as a valuable measure of pulmonary function in adults with late-onset Pompe disease. The investigators will adopt a strategy that includes comprehensive evaluations of respiratory volume, flow, and timing parameters during resting and loaded breathing. These evaluations will then be associated to the standard clinical measure of maximal inspiratory pressure, the static inspiratory muscle function, as well as magnetic resonance imaging of thoracic expansion and diaphragmatic descent at rest and with exertion. Outcomes in participants with late-onset Pompe disease will be contrasted to the function of age- and gender-matched control subjects. This approach will enable the investigators to evaluate the relationship between dynamic diaphragmatic function and respiratory motor function.
Detailed Description The following tests will be completed over a two-day period: Respiratory pressure tests, breathing test, magnetic resonance imaging and magnetic resonance spectroscopy (MRI and MRS).
Study Type Observational
Study Design Observational Model: Case-Control
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Subjects with late-onset Pompe disease, age 20-60 years, and age and gender-matched unaffected control subjects
Condition Pompe Disease
Intervention
  • Other: Thoracic MRI
    Imaging of the diaphragm will be completed during resting breathing, and then during a maximal voluntary ventilation maneuver.
  • Other: Spirometry
    Upright forced vital capacity (FVC)
  • Other: Inspiratory Load Compensation
    A spring-loaded pressure device will resist inspiration. The patient must generate the threshold inspiratory pressure to receive airflow. The changes in breathing timing, flow and volume will be recorded.
  • Other: Maximal Inspiratory Pressure
    Measure the maximal airflow on inhalation
    Other Name: MIP
  • Other: Resting Breathing Pattern
    The breathing pattern will be assessed when the subject is seated and in a relaxed state.
  • Other: Respiratory Muscle Endurance Test
    Evaluates the time limit that a participant can maintain ventilation while breathing with a submaximal inspiratory threshold load.
Study Groups/Cohorts
  • Pompe subjects
    These subjects will receive a thoracic MRI, spirometry, inspiratory load compensation, maximal inspiratory pressure, resting breathing pattern, respiratory muscle endurance test.
    Interventions:
    • Other: Thoracic MRI
    • Other: Spirometry
    • Other: Inspiratory Load Compensation
    • Other: Maximal Inspiratory Pressure
    • Other: Resting Breathing Pattern
    • Other: Respiratory Muscle Endurance Test
  • Control subjects
    These subjects will receive a thoracic MRI, spirometry, inspiratory load compensation, maximal inspiratory pressure, resting breathing pattern, respiratory muscle endurance test.
    Interventions:
    • Other: Thoracic MRI
    • Other: Spirometry
    • Other: Inspiratory Load Compensation
    • Other: Maximal Inspiratory Pressure
    • Other: Resting Breathing Pattern
    • Other: Respiratory Muscle Endurance Test
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 14, 2017)
19
Original Estimated Enrollment
 (submitted: January 29, 2015)
14
Estimated Study Completion Date December 2019
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Confirmed diagnosis of Pompe disease OR
  • Be an age and gender-matched unaffected control subject

Exclusion Criteria:

  • Pre-existing obstructive lung disease or asthma
  • Forced vital capacity (FVC) <30% or >80% of age/gender predicted values
  • Inability to travel to the study site
  • Requirement for positive pressure ventilator support when awake and upright
  • Participation in longitudinal studies that may independently alter lung function
  • Presence of any other chronic medical condition that, in the opinion of the investigator, would make the subject unsuitable for the study
Sex/Gender
Sexes Eligible for Study: All
Ages 20 Years to 60 Years   (Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Barbara K. Smith, PT, PhD 352-294-5315 bksmith@phhp.ufl.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02354664
Other Study ID Numbers IRB201400763
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party University of Florida
Study Sponsor University of Florida
Collaborators BioMarin Pharmaceutical
Investigators
Principal Investigator: Barbara K. Smith, PT, PhD University of Florida
PRS Account University of Florida
Verification Date December 2018