NCT03492346

Limb Girdle Muscular Dystrophy Type 2E Recruitment Study

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 25 (estimated)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 3 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

This study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial. Specifically, this trial is recruiting individuals who are suspected or have been confirmed to have Limb Girdle Muscular Dystrophy type 2E (LGMD2E).

Detailed description

This is a longitudinal observational study. It is a 24-month study with the possibility of extending the data time points. Visits will occur monthly. However, at the discretion of the PI, subjects may not be required to return monthly. These subjects may return at intervals ranging from 2 months to a max of 6 months apart. In the situation that the subjects would fall out of the inclusion criteria or not be eligible for the LGMD2E gene therapy trial, they will be given the opportunity to roll over into the Natural History for LGMD (IRB17-01086). If a subject is invited to screen for the gene therapy trial they will discontinue this trial. By being in this study, it is not a guarantee that subjects will be invited to screen for the LGMD2E gene therapy trial.

Conditions

• Limb-Girdle Muscular Dystrophy, Type 2E

Timeline

• Start date: 2018-03-28
• Primary completion: 2023-03-28
• Completion: 2023-03-28
• First posted: 2018-04-10
• Last updated: 2023-03-06

Locations

1 site across 1 country: United States