What is NNZ-2591 studying for Phelan-McDermid Syndrome?

NNZ-2591 is being studied to evaluate its effects on symptoms and behaviors in children with Phelan-McDermid Syndrome. This trial, sponsored by Neuren Pharmaceuticals Limited, compares NNZ-2591 to a placebo to understand its potential impact on this genetic condition. Participants receive either the study drug or placebo to assess changes over time.

Who may be eligible for this Phelan-McDermid Syndrome clinical trial?

Children aged 3 to 12 years with a confirmed diagnosis of Phelan-McDermid Syndrome and a documented SHANK3 genetic abnormality may be eligible. Participants must weigh at least 10 kg, not be actively losing skills, and have controlled seizure conditions. Please review eligibility criteria for more details.

Is there any cost to participate in this Phelan-McDermid Syndrome clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel or accommodation needs.

Actively Recruiting

Phase 3

Age: 3Years - 12Years

All Genders

ID07281079

Phase 3 Trial of Oral NNZ-2591 Versus Placebo in Children Aged 3 to 12 with Phelan-McDermid Syndrome

Led by Neuren Pharmaceuticals Limited · Updated on 2026-08-12

160

Participants Needed

Research Sites

2 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the efficacy and safety of NNZ-2591 compared to a placebo in children aged 3 to 12 years with Phelan-McDermid Syndrome, a genetic condition caused by an abnormality of the SHANK3 gene. This Phase 3, randomized, double-blind, placebo-controlled study aims to assess how NNZ-2591 affects symptoms and adaptive behaviors in pediatric participants. Participants first enter a 4-week screening period to confirm eligibility and assess symptom severity. Those eligible are randomly assigned to receive either NNZ-2591 or a matching placebo, both administered orally twice daily, over a 13-week treatment period. After treatment, there is a 2-week safety follow-up to monitor participants. During the study, participants undergo various assessments including the Phelan-McDermid Syndrome Assessment of Change PMSA-C and the Vineland Adaptive Behavior Scales-3 to measure communication and behavior changes. Caregiver impressions and clinical ratings are also collected. The total time commitment for participants is about 17 to 19 weeks, including screening, treatment, and follow-up periods.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 3Years - 12Years

All Genders

Eligibility Criteria

You may qualify if you...

Male or female pediatric participants with Phelan-McDermid syndrome aged 3 to 12 years at consent
Clinical diagnosis of Phelan-McDermid syndrome with documented SHANK3 genetic abnormality
Body weight of at least 10 kg at Screening
PMSA-S overall score of 3 or higher at Screening and Baseline visits
Not actively experiencing regression or loss of skills

You will not qualify if you...

Use of exclusionary medications or unstable treatment regimens as defined by the protocol
Current treatment with more than 3 psychotropic medications
Seizures not controlled on two or fewer anticonvulsant medications (excluding rescue medications)
Unstable doses of psychotropic or other chronic illness medications within 4 weeks before Screening
Any seizures within the past 6 months or more than one seizure in the past 12 months (except a single febrile seizure without rescue medication in past 6 months)
Abnormal liver function tests during Screening
Abnormal QT interval on Screening ECG as defined by the protocol

Research Team

Medical Information Lead

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