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Recruiting Phase 3

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

NCT07281079 · tracked via the Priya Life Science USA tracker
Sponsor
Neuren Pharmaceuticals Limited
Phase
Phase 3
Started
2025-11-12
Last updated
2026-09-28

Condition(s) studied

Phelan-McDermid Syndrome

Investigational drug(s) / intervention(s)

NNZ-2591 →Placebo

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

Sex
ALL
Min age
3 Years
Max age
12 Years
Healthy volunteers
No
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 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. 5. 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. 6. Abnormal liver function laboratory results during the Screening period, as defined by the protocol 7. Abnormal QT interval on Screening ECG as defined by the protocol.

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)

FacilityCityRegionStatus
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

Official registry record

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