Status:
RECRUITING
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
Lead Sponsor:
Astellas Gene Therapies
Conditions:
X-Linked Myotubular Myopathy
Eligibility:
MALE
Up to 17 years
Brief 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...
Eligibility Criteria
Inclusion
- Participant has a diagnosis of XLMTM resulting from a genetically confirmed mutation in the MTM1 gene based on genetic test reports.
- 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
- Participant is currently enrolled in an interventional study designed to treat XLMTM.
Key Trial Info
Start Date :
May 19 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 31 2027
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT06581146
Start Date
May 19 2025
End Date
May 31 2027
Last Update
January 7 2026
Active Locations (5)
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1
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
3
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104