NCT07281079

A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 160 (estimated)
• Sponsor: Neuren Pharmaceuticals Limited · Industry
• Sex: All
• Age: 3 Years – 12 Years
• Healthy volunteers: Not accepted

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.

Detailed description

After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome ages 3-12 years of age will enter the 4-week Screening Period and undergo assessments for eligibility, baseline characteristics and symptom severity. Once eligibility is confirmed, participants will be randomized in a 1:1 ratio to receive either orally administered NNZ-2591 or matching placebo during the 13-week Treatment Period. Subsequently, a 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.

Conditions

• Phelan-McDermid Syndrome

Interventions

Type	Name	Description
DRUG	NNZ-2591	The study drug will be administered twice daily orally.
DRUG	Placebo	The study drug will be administered twice daily orally.

Timeline

• Start date: 2025-11-12
• Primary completion: 2027-10-31
• Completion: 2027-11-15
• First posted: 2025-12-15
• Last updated: 2026-04-13

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study