Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
Study Status: Active, closed to enrollment
Patient Population:
o LGMD2A (CAPN3)
o LGMD2B (DYSF)
o LGMD2L (ANO5)
o LGMD1D (DNAJB6)
o LGMD2D (SGCA)
o LGMD2E (SGCB)
o LGMD2C (SGCD)
o LGMD2F (SGCG)
· Participation Duration: 12 months
· Study Summary:
This is a non-interventional study observing the natural progression of LGMD in patients over the course of 12 months. The study’s primary goal is to identify standardized clinical outcome assessments, in the form of functional measures, to hasten therapeutic development.
Defining Endpoints in Becker Muscular Dystrophy
· Study Status: Open to enrollment
· Patient Population: Becker Muscular Dystrophy (BMD)
· Participant Duration: 24 months
· Active Sites:
o Virginia Commonwealth University
o University of Kansas
o Nationwide Children’s Hospital
o University of California, Irvine
o University of Colorado
o University of Iowa
o Washington University in St. Louis
o Kennedy Krieger Institute
o University of Minnesota
o Leiden University Medical Center
o Newcastle University
· Upcoming Sites:
o NEuroMuscular Omnicentre Milano
o The University of Auckland
Study Summary:
This is a non-interventional study observing the natural progression of BMD in patients over the course of 24 months. The study’s primary goal is to assess the natural history of muscle function in these patients as well as the impact of BMD on a participant’s quality of life.
Trial Readiness and Endpoint Assessment in LGMDR1
Study Status: Open to enrollment
Patient Population: LGMDR1 (2A)
Participant Duration: 24 months
Active Sites:
Community Health Clinic Indiana
Leiden University
Nationwide Children’s Hospital
Newcastle University
University of California, Irvine
University of Colorado
University of Florida
University of Iowa
University of Kansas
University of Minnesota
Virginia Commonwealth University
Washington University in St. Louis
Study Summary:
This is a non-interventional study observing the natural progression of LGMDR1 in patients over the course of 24 months. The study’s primary goal is to validate the North Star Assessment for Dysferlinopathies (NSAD) as clinical outcome assessment for the LGMDR1 population to hasten therapeutic development.
Biomarker Development in LGMD2i
· Study Status: closed to enrollment
· Patient Population: LGMD2i (FKRP)
· Participation Duration: 12 months
· Active Sites:
o Virginia Commonwealth University
o University of Kansas
o Nationwide Children’s Hospital
o University of California, Irvine
o University of Colorado
o University of Iowa
o Washington University in St. Louis
o University of Florida
o Kennedy Krieger Institute
o Atrium Health
o University of Copenhagen