NNZ-2591 Treatment for Children with Phelan-McDermid Syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests whether a medication called NNZ-2591 can help children with Phelan-McDermid syndrome, a rare genetic condition that affects brain development and learning. Researchers want to see if this drug can improve how children with this condition develop and learn.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is 3–12 years old and was already part of a previous research study about Phelan-McDermid syndrome
- Your child completed all visits and tests in that earlier study
- Your child weighs at least 10 kg (about 22 pounds)
- Your child's condition is stable—not currently losing skills or abilities they once had
- Your child's liver function and heart rhythm are normal based on recent blood tests and heart monitoring
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a new medicine, NNZ-2591, to see if it helps children with Phelan-McDermid syndrome. It is for children aged 3 to 12 who have a specific genetic change (SHANK3) and weigh at least 10 kg.
This trial tests a gene therapy called JAG201 for children with a SHANK3 mutation or 22q13.3 deletion, which causes Phelan-McDermid syndrome. The goal is to see if it can help with development and daily function.
This study follows children who completed the RB001 gene therapy trial for Phelan-McDermid syndrome (caused by changes in the SHANK3 gene) to monitor their health and progress over a longer period of time.
This trial is testing a new medicine, NTX-253, to see how safe it is and how the body processes it. It is for healthy adults and adults with schizophrenia whose symptoms are stable on their current treatment.
This study tests a new medicine (S230815) given by lumbar puncture (spinal tap) to see if it helps reduce seizures in children with a specific genetic cause of epilepsy. It is for children aged 2 to 12 who have not responded well to other treatments.
This trial is testing an experimental drug (ION356) given by a lumbar puncture to see if it is safe and how it works in boys with Pelizaeus Merzbacher disease (PMD). The drug is designed to target the genetic cause of PMD.
Hear when a new Phelan-McDermid Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.