Study of development in children with CLN2 due to TPP1 changes
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study looks at how children’s development progresses in CLN2 disease caused by a specific enzyme problem (TPP1). It may help doctors better understand outcomes and guide future care for children with this diagnosis.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have CLN2 disease caused by a TPP1 enzyme deficiency
- Your diagnosis is confirmed by genetic testing showing disease-causing changes in the TPP1 gene
- If you are in a post-market study, you may still be able to join this study
- You must not be without both the CLN2 diagnosis and the TPP1 enzyme/genetic deficiency
- You likely cannot join if you are already enrolled in a larger multi-center clinical trial
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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