Stem cell transplant into the spinal fluid for rare brain diseases
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be between 1 week old and 21 years old
- Have a confirmed inherited metabolic brain disease on approved testing (examples include ALD, Batten disease, Krabbe, MLD, Hunter, Tay-Sachs, and more)
- Have signs the disease affects the nervous system (by symptoms, EEG/nerve tests, or brain MRI)
- Be well enough for treatment: heart, liver, kidneys, and lungs meet required lab/test levels
- Have a matching stored umbilical cord blood unit available for transplant
- Not have had certain prior transplants or gene/cell therapies in the last few years
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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