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Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 60

Last updated 2014-03-07

No results posted yet for this study

Summary

The purpose of this study is to understand the biochemistry of different types of Limb-Girdle Muscular Dystrophy (LGMD) and to determine appropriate outcome measures for future clinical treatment trials for LGMD. It is being conducted at two sites in the Cooperative International Neuromuscular Research Group (CINRG). It involves a one day clinical evaluation at a participating institution that will take approximately four to six hours, and will involve strength testing and muscle functional testing by a physical therapist, an evaluation by a physician, pulmonary function testing, a complete cardiac evaluation with electrocardiogram (ECG or EKG) and echocardiogram (Echo), and involve two blood draws, one before the evaluation and one after the evaluation is complete. During the visit, the participant will be asked to fill out a couple of questionnaires asking questions about quality of life and activity limitations, as well as his/her understanding of their diagnosis with regards to etiology (or cause of their muscle disorder), genetics, and inheritance of their muscle disorder.

Conditions

  • Becker Muscular Dystrophy
  • Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency)
  • Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency)
  • Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)

Sponsors & Collaborators

  • Carolinas Medical Center lead study site

    collaborator UNKNOWN

  • Cooperative International Neuromuscular Research Group

    lead NETWORK

Principal Investigators

  • Carolina Tesi-Rocha, M.D. · Cooperative International Neuromuscular Research Group

  • Susan Sparks, M.D., Ph.D. · Levine Children's Hospital

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2009-04-30
Primary Completion
2013-12-31
Completion
2013-12-31

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 NCT00893334 on ClinicalTrials.gov