Study on eli-cel for cerebral adrenoleukodystrophy
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study follows people with cerebral adrenoleukodystrophy (CALD) who have been treated with eli-cel, a gene therapy. It tracks their long-term health to see how well the treatment works in everyday care.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been treated with eli-cel at a US center that takes part in this study.
- You must agree to participate in the CIBMTR registry and this study by signing a consent form.
- Your follow-up care must be with a US doctor who can provide study data.
- For the advanced group: your MRI before treatment must show a certain brain change (Loes score 4.5–9.0 with gadolinium enhancement) and you had mild or no symptoms (NFS 0 or 1).
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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