Trial readiness for dystroglycan-related muscle conditions
Part of Bones, joints & muscles, Brain & nervous system, Eyes & vision, Genetic & congenital clinical trials.
This trial is for people who have signs that they may have a dystroglycanopathy—a group of muscle conditions. It helps study how ready participants are for care and possibly future treatments by using lab and genetic/pathology information to confirm the condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your blood test shows elevated creatine kinase (CK).
- You have evidence of a dystroglycanopathy from one of these: muscle biopsy/pathology, a known gene mutation, or a lab test showing abnormal alpha-dystroglycan in grown skin-cell samples (fibroblasts).
- You can be any age, including children.
- Males and females are both welcome.
- You should have some muscle weakness, but be otherwise in relatively good health.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study collects blood and, for some people, muscle tissue to look for signs (biomarkers) that can help track and better understand myotonic dystrophy. You may help by providing samples and basic muscle function testing, depending on your age and condition type.
This trial collects health information from patients with certain inherited (genetic) muscle diseases and from their families. It may help researchers better understand the disease and improve care by learning what symptoms and treatments patients experience over time.
This study measures physical and lab “health markers” in people with myotonic dystrophy type 1 (DM1) to better understand how the disease changes over time. Some participants may also have a muscle biopsy to study tissue differences.
This study tracks changes in muscle strength and breathing over time in people with certain types of muscular dystrophy. It helps researchers develop better tests to measure how the disease progresses.
This study looks at how to best measure muscle health and function in children with myotonic dystrophy. It may help prepare for future treatment trials.
This study uses MRI scans and blood or other biomarkers to better understand muscular dystrophy in people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). It may help researchers track disease changes and identify measurable markers over time.
Hear when a new Muscular Dystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.