Clinical Trial Readiness for the Dystroglycanopathies (NCT00313677) | Clinical Trial Compass
RecruitingNot Applicable
Clinical Trial Readiness for the Dystroglycanopathies
United States190 participantsStarted 2006-04
Plain-language 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.
Who can participate
SexALL
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Inclusion Criteria:
* 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 Criteria:
* There are no exclusion criteria.
What they're measuring
1
10 meter walk
Timeframe: through study completion, an average of 1 yea