Phelan-McDermid Syndrome is a rare disorder affecting development, often linked to issues with a gene named SHANK3. This study tests a medicine called NNZ-2591 to see if it helps children with this syndrome. In a Phase 3 study, researchers carefully compare the medicine to a placebo, which looks like the medicine but doesn’t have any active ingredients.
Children ages 3-12 with the syndrome can join. First, there's a 4-week Screening Period to check if they qualify. Then, they will take either NNZ-2591 or the placebo for 13 weeks. After treatment, there will be a 2-week Safety Follow-up to monitor any side effects.
- This study lasts a total of 19 weeks, including screening and follow-up.
- Participants will be randomly assigned to receive either the medication or placebo.
- Children on certain medications or with certain health issues may not qualify.
By participating, families help researchers understand if NNZ-2591 can be a safe and effective treatment for Phelan-McDermid Syndrome.