Study of inherited leukodystrophy symptoms and outcomes
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study follows people with inherited leukodystrophies to better understand how the condition is diagnosed and how it changes over time. It may help doctors improve testing and care planning by collecting clinical and imaging information.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have an inherited leukodystrophy, based on symptoms, brain scans, and/or test results
- You can travel to the leukodystrophy clinic at Primary Children’s Hospital in Salt Lake City, Utah
- You can take part in a general physical exam and a neurological (brain/nerve) exam
- You can be evaluated at University of Utah Hospital or Primary Children’s Hospital
- You agree to sign the study consent form
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 brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
This trial follows people with genetic neurodegenerative (brain-wasting) disorders over time to better understand how the condition changes. The goal is to learn patterns that could help future treatments or care plans.
This study looks at immune cells in blood to better understand rare brain conditions caused by genetic changes. It may help researchers understand why symptoms happen and how the immune system is involved.
This Phase 1 trial tests a transplant using special donated stem-cell–like cells placed into the spinal fluid to treat certain inherited brain diseases. It is mainly designed to check safety, and it may offer benefit by targeting brain and nerve damage.
This study follows women with a genetic condition called X-linked adrenoleukodystrophy (ALD) over time to see how the disease progresses. It includes annual check-ups and MRI scans of the brain and spinal cord to monitor changes.
This study looks at children with inherited muscle diseases (not Duchenne muscular dystrophy) to understand their symptoms and muscle function better. It may help doctors identify what type of muscle disease a child has and how it affects them day-to-day.
Hear when a new Leukodystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.