RecruitingPhase 3NCT07281079

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

Sponsor

Neuren Pharmaceuticals Limited

Enrollment

160 participants

Start Date

Nov 12, 2025

Study Type

INTERVENTIONAL

Conditions

Phelan-McDermid Syndrome

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

Min Age: 3 YearsMax Age: 12 Years

Inclusion Criteria5

Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent.
Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3.
Body weight ≥ 10 kg at Screening.
Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits.
Not actively undergoing regression or loss of skills.

Exclusion Criteria7

Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol.
Current treatment with more than 3 allowable psychotropic medications.
Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications).
Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regimen that have not been stable for at least 4 weeks before Screening. If the treatment was discontinued, the discontinuation must have occurred no fewer than 2 weeks before the start of Screening.
Any intercurrent seizures in the past 6 months and /or more than 1 seizure in the past 12 months. •A single febrile seizure in the 6 months prior to screening is allowable if no rescue medication was required.
Abnormal liver function laboratory results during the Screening period, as defined by the protocol
Abnormal QT interval on Screening ECG as defined by the protocol.

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