PLX-200 for children with CLN2, CLN3, Sandhoff, or Krabbe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests an experimental drug called PLX-200 for children with certain rare brain diseases. It aims to see if the drug is safe, tolerable, and helpful for slowing the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of CLN2, CLN3, Sandhoff disease, or Krabbe disease
- Your child must be between 2 and 15 years old
- Your child must not need a ventilator (except for sleep support like CPAP machines)
- Your child must not have had a stem cell transplant or gene therapy
- Your child should not have uncontrolled seizures (more than 4 major seizures per month)
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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