Status:
COMPLETED
A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)
Lead Sponsor:
Astellas Gene Therapies
Conditions:
Males With X-linked Myotubular Myopathy (XLMTM)
Eligibility:
MALE
Brief Summary
This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical manif...
Eligibility Criteria
Inclusion
- Patient diagnosed with XLMTM resulting from a confirmed mutation in the MTM1 gene, or a combination of XLMTM genetically confirmed family history and muscle biopsy
- Patient is male
- Access to available medical records for each patient
- Signed informed consent by the parent(s) or legal guardians and/or assent by the patient (when applicable), unless the associated IRB provides an appropriate consent waiver
Exclusion
- Patient data after participation in an interventional study designed to treat XLMTM (patient data prior to participation in an interventional study may be included)
Key Trial Info
Start Date :
September 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 1 2019
Estimated Enrollment :
161 Patients enrolled
Trial Details
Trial ID
NCT02231697
Start Date
September 1 2014
End Date
December 1 2019
Last Update
July 29 2022
Active Locations (8)
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1
Cure CMD
San Pedro, California, United States, 90732
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
University of Florida - Gainesville, Children's Research Institute
Gainesville, Florida, United States, 32610
4
Lurie Children's Hospital
Chicago, Illinois, United States, 60611