Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)
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ClinicalTrials.gov Identifier: NCT02453152 |
Recruitment Status :
Completed
First Posted : May 25, 2015
Last Update Posted : February 15, 2021
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Sponsor:
University of Florida
Collaborator:
Audentes Therapeutics
Information provided by (Responsible Party):
University of Florida
Tracking Information | |||||
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First Submitted Date | May 18, 2015 | ||||
First Posted Date | May 25, 2015 | ||||
Last Update Posted Date | February 15, 2021 | ||||
Study Start Date | October 2015 | ||||
Actual Primary Completion Date | July 31, 2019 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures |
Change in baseline visit off-ventilator tolerance at 6 month visit and 12 month visit. [ Time Frame: Change in baseline visit, at 6 month visit and 12 month visit ] | ||||
Original Primary Outcome Measures | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures |
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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 | Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM) | ||||
Official Title | Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM) | ||||
Brief Summary | This study is a longitudinal study evaluating the severity and progression of respiratory muscle function in patients with X-Linked Myotubular Myopathy (XLMTM) aged 0-14. | ||||
Detailed Description | Subjects aged 0-7 will be evaluated every six months for a total of three evaluations (Baseline, Six-Month Visit, Twelve-Month Visit). Subjects aged 8-14 will be evaluated every twelve months for a total of two evaluations (Baseline, Twelve-Month Visit). Evaluations include medical history, physical exam, respiratory muscle tests, a qualitative interview related to the child's function and use of respiratory aids, and quality of life assessments. | ||||
Study Type | Observational | ||||
Study Design | Observational Model: Cohort Time Perspective: Prospective |
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Target Follow-Up Duration | Not Provided | ||||
Biospecimen | Not Provided | ||||
Sampling Method | Non-Probability Sample | ||||
Study Population | Males with geneticially confirmed XLMTM | ||||
Condition | X-linked Myotubular Myopathy | ||||
Intervention |
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Study Groups/Cohorts | 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
Interventions:
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status | Completed | ||||
Actual Enrollment |
28 | ||||
Original Estimated Enrollment |
12 | ||||
Actual Study Completion Date | July 31, 2019 | ||||
Actual Primary Completion Date | July 31, 2019 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender |
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Ages | up to 14 Years (Child) | ||||
Accepts Healthy Volunteers | No | ||||
Contacts | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number | NCT02453152 | ||||
Other Study ID Numbers | IRB201500379 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement | Not Provided | ||||
Current Responsible Party | University of Florida | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor | University of Florida | ||||
Original Study Sponsor | Same as current | ||||
Collaborators | Audentes Therapeutics | ||||
Investigators |
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PRS Account | University of Florida | ||||
Verification Date | February 2021 |