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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Phelan-McDermid Syndrome

In brief

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.

Key facts

Study ID

NCT07281079

Run by

Neuren Pharmaceuticals Limited

People needed

160

Starts

2025-11-12

Expected to finish

2027-11-15

Last updated by the study team

2026-07-07

Who can join

Age: 3 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Neuren PMS-301 Site#122 — Glendale, California, United States (enrolling)
• Neuren PMS-301 Site #112 — Palo Alto, California, United States (enrolling)
• Neuren PMS-301 Site#103 — San Diego, California, United States (enrolling)
• Neuren PMS-301 Site#111 — San Rafael, California, United States (enrolling)
• Neuren PMS-301 Site#102 — Chicago, Illinois, United States (enrolling)
• Neuren PMS-301 Site#109 — Chevy Chase, Maryland, United States (enrolling)
• Neuren PMS-301 Site#106 — Brookline, Massachusetts, United States (enrolling)
• Neuren PMS-301 Site#104 — Lexington, Massachusetts, United States (enrolling)
• Neuren PMS-301 Site#101 — New York, New York, United States (enrolling)
• Neuren PMS-301 Site#108 — Cincinnati, Ohio, United States (enrolling)
• Neuren PMS-301 Site#115 — Houston, Texas, United States (enrolling)
• Neuren PMS-301 Site#201 — Toronto, Ontario, Canada (enrolling)

Full record on ClinicalTrials.gov

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