Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)
NCT ID: NCT02453152
Last Updated: 2021-02-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
28 participants
OBSERVATIONAL
2015-10-31
2019-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Males with X-linked myotubular myopathy
History and physical, Tidal breathing, Maximal respiratory pressures, Peak cough flow, Pediatric Evaluation of Disability Inventory, PedsQL Multidimensional Fatigue Scale, Review of ventilation requirements
History and physical
Subjects will undergo a physical exam and medical history review, including review of genetic test results.
Tidal breathing
Subjects' breathing patterns will be evaluated at rest and at reduced/eliminated ventilator support.
Maximal respiratory pressures
Subjects' strongest inspiratory and expiratory pressures will be measured.
Peak cough flow
Subjects' strongest coughs will be measured.
Pediatric Evaluation of Disability Inventory
Assesses the functional capabilities of children with disabilities.
PedsQL Multidimensional Fatigue Scale
Assesses general fatigue, sleep/rest fatigue, and cognitive fatigue.
Review of ventilation requirements
Subjects's use of mechanical ventilation reviewed by the study team.
Interventions
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History and physical
Subjects will undergo a physical exam and medical history review, including review of genetic test results.
Tidal breathing
Subjects' breathing patterns will be evaluated at rest and at reduced/eliminated ventilator support.
Maximal respiratory pressures
Subjects' strongest inspiratory and expiratory pressures will be measured.
Peak cough flow
Subjects' strongest coughs will be measured.
Pediatric Evaluation of Disability Inventory
Assesses the functional capabilities of children with disabilities.
PedsQL Multidimensional Fatigue Scale
Assesses general fatigue, sleep/rest fatigue, and cognitive fatigue.
Review of ventilation requirements
Subjects's use of mechanical ventilation reviewed by the study team.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patients who are between 0 and 14 years of age.
Exclusion Criteria
* Patients unable to travel to the site for the study.
* Patients participating in an interventional treatment study for XLMTM at the time of enrollment.
* Patients who are unable to complete study procedures.
* Patients who have a condition that, in the opinion of the investigator, would make participation in this study unsafe.
14 Years
MALE
No
Sponsors
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Audentes Therapeutics
INDUSTRY
University of Florida
OTHER
Responsible Party
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Principal Investigators
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Barbara K Smith, PT, PhD
Role: PRINCIPAL_INVESTIGATOR
University of Florida
Locations
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University of Florida
Gainesville, Florida, United States
Countries
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Other Identifiers
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IRB201500379
Identifier Type: -
Identifier Source: org_study_id
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