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Defining Clinical Endpoints in LGMD

Status: 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

Age: Not specified
Healthy Volunteers:
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)
Conditions:

Rare Diseases

Keywords:

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

Contact(s): Allison Johnston - joh21779@umn.edu
Principal Investigator: Peter Kang
IRB Number: SITE00001632
System ID: 39368
See this study on ClinicalTrials.gov

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