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Defining Clinical Endpoints in LGMD

Recruiting

Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

I'm interested

Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• 4 to 65 years of age
• diagnosis of Muscular Dystrophy with weakness in either a limb-girdle pattern, or in a arm or leg
• confirmed mutation in ANO5, CAPN3, DYSF, DNAJB6 or SGCA-G.
Exclusion Criteria:

• bleeding disorder, platelet count less than 50,000, or currently taking an anticoagulant.
• women who are pregnant
• other illness that would interfere clinical trial (study staff will review)

Rare Diseases

Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy (LGMD), Muscular Dystrophy

Allison Johnston - joh21779@umn.edu
Peter Kang
SITE00001632
39368
See this study on ClinicalTrials.gov

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