Status:
RECRUITING
Clinical Trial Readiness for the Dystroglycanopathies
Lead Sponsor:
Katherine Mathews
Collaborating Sponsors:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Muscular Dystrophy
Eligibility:
All Genders
Brief Summary
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials.
Detailed Description
Muscular dystrophies are a diverse group of inherited disorders characterized by progressive muscle weakness and wasting. The disorders are caused by mutations, or changes, in genes. Genes are tiny pi...
Eligibility Criteria
Inclusion
- Elevated CK (creatine kinase)
- Evidence of a dystroglycanopathy as determined by review of muscle pathology OR documented mutation in one of the known genes OR abnormal alpha-dystroglycan glycosylation in cultured fibroblasts
- Dystroglycanopathies are predicted to affect all racial and ethnic backgrounds, and all patients with dystroglycanopathies will be eligible for participation.
- Participants may be of any age, including children, and males and females will be recruited equally.
- Patients will have varying degrees of muscular weakness, but otherwise should be in relatively good health.
Exclusion
- There are no exclusion criteria.
Key Trial Info
Start Date :
April 1 2006
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
July 1 2030
Estimated Enrollment :
190 Patients enrolled
Trial Details
Trial ID
NCT00313677
Start Date
April 1 2006
End Date
July 1 2030
Last Update
July 30 2025
Active Locations (1)
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1
University of Iowa, 200 Hawkins Drive
Iowa City, Iowa, United States, 52242