A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Condition(s) studied
Investigational drug(s) / intervention(s)
NNZ-2591: The study drug will be administered twice daily orally.
Placebo: The study drug will be administered twice daily orally.
Study 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
Primary outcome measure(s)
- Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. — Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. The PMSA-C scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse. - Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score. — Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in the Vineland Adaptive Behavior Scales-3, Interview version (Vineland-3) receptive communication subdomain raw score. A higher raw score for the receptive communication subdomain indicates better adaptive behavior.
Trial sites (18)
| Facility | City | Region | Status |
|---|---|---|---|
| Neuren PMS-301 Site #116 | Birmingham | Alabama | Recruiting |
| Neuren PMS-301 Site#122 | Glendale | California | Recruiting |
| Neuren PMS-301 Site #112 | Palo Alto | California | Recruiting |
| Neuren PMS-301 Site#103 | San Diego | California | Recruiting |
| Neuren PMS-301 Site#111 | San Rafael | California | Recruiting |
| Neuren PMS-301 Site #107 | Washington D.C. | District of Columbia | Recruiting |
| Neuren PMS-301 Site #105 | Atlanta | Georgia | Recruiting |
| Neuren PMS-301 Site#102 | Chicago | Illinois | Recruiting |
| Neuren PMS-301 Site#109 | Chevy Chase | Maryland | Recruiting |
| Neuren PMS-301 Site#106 | Brookline | Massachusetts | Recruiting |
| Neuren PMS-301 Site#104 | Lexington | Massachusetts | Recruiting |
| Neuren PMS-301 Site #117 | Ann Arbor | Michigan | Recruiting |
| Neuren PMS-301 Site#101 | New York | New York | Recruiting |
| Neuren PMS-301 Site#108 | Cincinnati | Ohio | Recruiting |
| Neuren PMS-301 Site#110 | Columbus | Ohio | Recruiting |
| Neuren PMS-301 Site#115 | Houston | Texas | Recruiting |
| Neuren PMS-301 Site #113 | Charlottesville | Virginia | Recruiting |
| Neuren PMS-301 Site#201 | Toronto | Ontario | Recruiting |
More Neuren Pharmaceuticals Limited trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07281079 on ClinicalTrials.gov ↗ ← All trials in the USA