A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
NCT ID: NCT06581146
Last Updated: 2026-01-07
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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RECRUITING
50 participants
OBSERVATIONAL
2025-05-19
2027-05-31
Brief Summary
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Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health. Bile is a liquid made in the liver that helps digest fat. Ongoing liver health checks may help with the routine care of people with XLMTM.
There is a need to understand liver problems that develop in individuals with XLMTM over time. The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study.
This study is about collecting information only. This is known as an observational study. The individual's doctor decides on treatment, not the study sponsor (Astellas).
In this study, boys under 18 diagnosed with XLMTM will be followed for about 1 year. The health of their liver and gallbladder will be checked about every 6 weeks. This can be done at home, if preferred. A scan called a Fibroscan (also known as transient elastography) will check for signs of scarring in the liver (fibrosis) and the build-up of lipids. It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study.
This study will help understand liver, gallbladder, and bile duct issues in individuals with XLMTM over time. The goal is to improve their care and provide information to use in future clinical studies.
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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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Participants with XLMTM
Pediatric and adolescent participants with XLMTM.
No Intervention
No investigational drug will be administered to participants in this study.
Interventions
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No Intervention
No investigational drug will be administered to participants in this study.
Eligibility Criteria
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Inclusion Criteria
* Participant requires some mechanical ventilatory support (e.g., ranging from 24 hours per day full-time mechanical ventilation, to non-invasive support such as continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) during sleeping hours)
* Participant (as applicable) and/or parent(s)/carer is willing to comply with the recommended schedule of assessments.
Exclusion Criteria
17 Years
MALE
No
Sponsors
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Astellas Gene Therapies
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Astellas Gene Therapies
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Countries
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Central Contacts
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Other Identifiers
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1600-MA-3536
Identifier Type: -
Identifier Source: org_study_id
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