Experimental ASO treatment for TUBB4A-related leukodystrophy
Part of Genetic & congenital clinical trials.
This trial tests a single patient's response to an experimental genetic treatment (called an ASO) for a rare brain disease caused by changes in the TUBB4A gene. The goal is to see if the treatment can improve symptoms related to the condition H-ABC.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must have a confirmed genetic change (mutation) in the TUBB4A gene.
- The diagnosis should match a brain condition called H-ABC (Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum).
- You must be able to travel to the study site and follow up regularly.
- You must be willing to have a lumbar puncture (a needle in the lower back to collect fluid).
- You cannot have taken any other experimental drug within the last few weeks (or longer, depending on the drug).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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