Study of PLX-200 for children with certain lysosomal storage diseases
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests an investigational drug called PLX-200 in children with four rare lysosomal storage diseases. The goal is to see if it is safe and might slow down the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 2 and 15 years old.
- They must have a confirmed diagnosis (by genetic test) of one of these four diseases: CLN2, CLN3, Sandhoff disease, or Krabbe disease.
- Parents or guardians must be willing to follow the study schedule and give permission. Older children must also agree to participate.
- Your child cannot have any other unrelated brain or nerve disease.
- They cannot need a breathing machine (except for sleep apnea machines like CPAP or BiPAP).
- They cannot have severe liver, kidney, or gallbladder problems, or significant anemia.
- They cannot be on certain medications (like statins or fibrates), and must have stopped them for at least 2 weeks (or 5 half-lives) before starting.
- They cannot have had stem cell or gene therapy at any time.
- If your child has seizures, they must be controlled (fewer than 4 generalized seizures per month, and no recent status epilepticus).
- They cannot have serious heart rhythm problems or severe brain abnormalities like hydrocephalus or a shunt.
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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