Studying different forms of childhood brain-wasting disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study looks at people with CLN3 and other related forms of NCL (a childhood brain-wasting illness) to better describe symptoms and disease changes over time. You may be followed with regular check-ins, medical records, and sometimes NIH visits or sample collection.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be older than 1 week and have a diagnosis of CLN3 or another NCL type, or be eligible through a family history (depending on the sub-study).
- For CLN3/NCL diagnosis, genetic testing often needs two disease-causing gene changes, or one gene change plus symptoms and/or specific lab (electron microscope) findings.
- If you are in the main study, be able to travel to the NIH (or otherwise participate as described).
- Be able to follow study steps and have no health issues that would make participation unsafe.
- If you are pregnant, you cannot join the main study.
- Sub-study B (and A/B as stated) may exclude adults over 18 who are unaffected but have cognitive impairments.
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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