Lentiviral gene therapy for childhood cerebral X-ALD
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial studies an experimental gene therapy given through infusion (IV) and into the spinal fluid (injection) for people with X-ALD affecting the brain. It aims to improve or slow down damage in the nervous system, and you must be able to do MRI scans and meet specific health and infection requirements.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be diagnosed with X-ALD and be at least 1 year old
- Have brain involvement from ALD, based on a blood test called VLCFA levels
- Have a recent brain MRI available for study doctors to review
- Have a neurological function score of 1 or higher
- Be able to give informed consent (or have a parent/guardian consent for a child)
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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