A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome (NCT07281079) | Clinical Trial Compass
RecruitingPhase 3
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
United States160 participantsStarted 2025-11-12
Plain-language 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.
Who can participate
Age range3 Years – 12 Years
SexALL
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Inclusion criteria
✓. 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 criteria
✕. 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.
What they're measuring
1
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.
Timeframe: Week 13
2
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.