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Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

NCT03488784 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2025-08-14

No results posted yet for this study

Summary

This is an observational study, no drug (marketed or investigational) will be provided as part of the study, and the study procedures will have no impact on the medical care delivered to patients participating in this study. The overall study data collection period is planned to last up to 5 years with assessments occurring at baseline, and every 6 months thereafter for a total period of 3 years.

Medical records for enrolled patients will be abstracted at baseline and annually to obtain clinical information, and data will be recorded for the study. Eligible patients will be asked to provide informed consent and to complete semi-annual patient surveys and functional assessments. The patient surveys will include selected PRO instrument(s) along with additional questions to characterize the patient's perception of disease.

Conditions

  • Limb-Girdle Muscular Dystrophy Type 2A
  • Limb-Girdle Muscular Dystrophy, Type 2E

Sponsors & Collaborators

  • Lindsay Alfano

    lead OTHER

Principal Investigators

  • Linda Lowes, PT PhD · Nationwide Children's Hospital

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-11-29
Primary Completion
2025-08-11
Completion
2025-08-11

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03488784 on ClinicalTrials.gov