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NCT06581146 · ClinicalTrials.gov registry record
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
A clinical trial of X-Linked Myotubular Myopathy, sponsored by Astellas Gene Therapies.
- Recruiting
- Registry status
- 50
- Enrollment target
- 5
- Study locations
NCT06581146: Recruiting study of X-Linked Myotubular Myopathy, sponsored by Astellas Gene Therapies.
NCT06581146 is a study of X-Linked Myotubular Myopathy that is actively recruiting participants, run by Astellas Gene Therapies. The registered enrollment target is 50 participants, above the 23-participant average among 3 other X-Linked Myotubular Myopathy trials with a reported enrollment target (117% higher). The trial reports 5 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06581146, a study of X-Linked Myotubular Myopathy, is actively recruiting participants, sponsored by Astellas Gene Therapies.
- RECRUITING
- Registry status
- 50 participants
- Enrollment target
- 5
- Study locations
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.
Primary Outcome
Calculated as the number of new cases of cholestasis over 48 weeks divided by total duration of follow-up for enrolled participants.
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 |
What NCT06581146 shows while recruiting
NCT06581146 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target, above the 23-participant average among 3 other X-Linked Myotubular Myopathy trials with a reported enrollment target (117% higher).
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.
NCT06581146 lists 5 locations in 4 states (Pennsylvania, Illinois, Massachusetts).
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.
What interventions are being tested in trial NCT06581146?
The interventions under investigation include: No Intervention (OTHER).
Who is sponsoring clinical trial NCT06581146?
This trial is sponsored by Astellas Gene Therapies, which has 7 total clinical trials registered on ClinicalTrials.gov.
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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