Actively Recruiting
Clinical Trial Readiness for the Dystroglycanopathies
Led by Katherine Mathews · Updated on 2025-07-30
190
Participants Needed
1
Research Sites
1265 weeks
Total Duration
On this page
Sponsors
K
Katherine Mathews
Lead Sponsor
N
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Clinical Trial Readiness for the Dystroglycanopathies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Elevated CK (creatine kinase)
- Evidence of a dystroglycanopathy confirmed by muscle pathology review, documented mutation in a known gene, or abnormal alpha-dystroglycan glycosylation in cultured fibroblasts
- Participants of any age and all racial and ethnic backgrounds are eligible
- Both males and females will be recruited equally
- Participants may have varying degrees of muscular weakness but should be in relatively good health
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of Iowa, 200 Hawkins Drive
Iowa City, Iowa, United States, 52242
Actively Recruiting
Research Team
C
Carrie Stephan, R.N. M.A.
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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