New study aims to uncover hidden liver risks in rare muscle disease
NCT ID NCT06581146
First seen Jun 25, 2026 · Last updated Jul 17, 2026 · Updated 4 times
Summary
This study follows about 50 boys under 18 with X-linked myotubular myopathy (XLMTM), a rare genetic muscle condition, to see how often they develop liver problems like cholestasis. No drugs are given—researchers simply collect health data, including liver scans, over one year. The goal is to better understand liver and gallbladder issues in XLMTM to improve care and guide future studies.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this study could help doctors better monitor and manage liver issues in boys with XLMTM, potentially improving their routine care.
- What could go wrong
- This is an observational study with no treatment being tested, so it won't directly change outcomes. It only follows 50 boys for one year, which may not capture all liver problems.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
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Site CA15001
RECRUITINGToronto, Ontario, Canada
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Site GB44003
RECRUITINGLondon, United Kingdom
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Site GB44005
RECRUITINGOxford, United Kingdom
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Site GB44006
RECRUITINGLeeds, United Kingdom
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UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
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