Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

Completed

Conditions studied: X-linked Myotubular Myopathy

In brief

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.

Key facts

Study ID

NCT02453152

Run by

University of Florida

People needed

28

Starts

2015-10-01

Expected to finish

2019-07-31

Last updated by the study team

2021-02-15

Who can join

Age: any, up to 14. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Patients who have centronuclear myopathy resulting from an MTM1 genetic mutation.
• Patients who are between 0 and 14 years of age.

You may not qualify if…

• Patients without a confirmed genetic mutation.
• 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.

Where it is running

• University of Florida — Gainesville, Florida, United States

Full record on ClinicalTrials.gov

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