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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
NCT06581146 · View on ClinicalTrials.gov ↗
Study Summary
XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition. 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.
Conditions Studied
Interventions
- OTHER No Intervention
Study Locations (5)
Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia
- UPMC Children's Hospital of Pittsburgh — Pittsburgh
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Massachusetts
- Boston Children's Hospital — Boston
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2025-05-19 |
| Est. Completion | 2027-05-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06581146
The ClinicalTrials.gov registry entry for NCT06581146 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 50 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Astellas Gene Therapies, which has 36 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with X-Linked Myotubular Myopathy appearing as the primary indexed condition, and to 1 intervention — of which No Intervention is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06581146 reports 5 study locations spanning 4 distinct geographic areas — top geographies include Pennsylvania, Illinois, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06581146 about?
NCT06581146 is a clinical study titled "A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition". XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent furthe...
What is the current status of trial NCT06581146?
This trial is currently recruiting. The enrollment target is 50 participants. The study started on 2025-05-19. Estimated completion is 2027-05-31.
What conditions does trial NCT06581146 study?
This clinical trial studies the following conditions: X-Linked Myotubular Myopathy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06581146?
The interventions under investigation include: No Intervention (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06581146?
This trial is sponsored by Astellas Gene Therapies, which has 36 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06581146 being conducted?
This trial has 5 study locations across Illinois, Massachusetts, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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