Gene therapy for ataxia-telangiectasia with specific ATM mutation
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism, Immune system & allergy clinical trials.
This trial tests a new genetic medicine designed for people with A-T who have a specific ATM gene change. It aims to see if the treatment is safe and if it can improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have classic ataxia-telangiectasia (A-T) confirmed by genetic testing
- You must have a specific ATM gene change called c.7865C>T
- You must also have a second ATM gene change that causes A-T
- You cannot have bleeding problems, brain pressure issues, serious heart or lung problems, or infections near your lower back
- You must be healthy enough for a lumbar puncture
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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