A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Trial Parameters
Brief Summary
This Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compared to placebo in pediatric participants with Phelan- McDermid Syndrome.
Eligibility Criteria
Inclusion Criteria: 1. Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent. 2. Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3. 3. Body weight ≥ 10 kg at Screening. 4. Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits. 5. Not actively undergoing regression or loss of skills. Exclusion Criteria: 1. Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol. 2. Current treatment with more than 3 allowable psychotropic medications. 3. Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications). 4. Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regime