Stem cell transplant for nerve damage from homocysteine remethylation disorders
This trial tests using your own blood stem cells to treat nerve damage caused by certain inherited conditions that disrupt homocysteine metabolism. It aims to slow or stop the disease from getting worse when standard treatments like vitamins have not worked well enough.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 55 years old.
- You have a confirmed inherited homocysteine remethylation disorder with nerve-related symptoms like trouble walking, balance problems, memory issues, or brain changes.
- You have tried standard therapy (folic acid, vitamin B12, betaine) and your symptoms did not improve enough.
- Your homocysteine level remains very high (above 50 umol/L) despite treatment.
- Your medical team agrees that there are no other effective treatments and your condition is likely to get worse.
- You have not had a stem cell transplant before and you do not have serious organ, infection, cancer, or psychiatric issues that would make the trial unsafe.
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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