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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
Led by Neuren Pharmaceuticals Limited · Updated on 2026-05-29
160
Participants Needed
7
Research Sites
2 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of NNZ-2591 compared to a placebo in children aged 3 to 12 years with Phelan-McDermid Syndrome, a rare genetic condition caused by abnormalities in the SHANK3 gene. This Phase 3, randomized, double-blind, placebo-controlled study includes multiple centers to ensure broad participation and reliable results. After informed consent, participants will enter a 4-week screening period to confirm eligibility and assess baseline symptoms. Those who qualify will be randomly assigned to receive either NNZ-2591 or a matching placebo, both taken orally twice daily, during a 13-week treatment period. Following treatment, there will be a 2-week safety follow-up to monitor participants. During the study, children will undergo various assessments including the Phelan-McDermid Syndrome Assessment of Change and the Vineland Adaptive Behavior Scales to evaluate communication and behavior changes. Caregivers will provide impressions of change, and researchers will monitor safety through clinical evaluations. The total participation time per child is approximately 17 to 19 weeks.
CONDITIONS
Brief Title
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female pediatric participants with Phelan-McDermid syndrome aged 3 to 12 years at the time of consent
- Clinical diagnosis of Phelan-McDermid syndrome with a documented SHANK3 genetic abnormality
- Body weight of at least 10 kilograms at screening
- PMSA-S overall score of 3 or higher at Screening and Baseline visits
- Not currently experiencing regression or loss of skills
You will not qualify if you...
- Use of exclusionary medications or unstable treatment regimens as specified by the protocol
- Treatment with more than 3 allowable psychotropic medications
- Participants with seizures must be controlled on no more than 2 anticonvulsant medications, excluding rescue medications
- Psychotropic or chronic illness medications not stable for at least 4 weeks before screening or discontinued less than 2 weeks before screening
- Any seizures in the past 6 months or more than one seizure in the past 12 months (a single febrile seizure without rescue medication in the past 6 months is allowed)
- Abnormal liver function tests during screening as defined by the protocol
- Abnormal QT interval on screening ECG as defined by the protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 4 weeks
Participants are screened for eligibility to participate in the trial.
Assessments for eligibility, baseline characteristics, and symptom severity occur during this period.
Duration - 13 weeks
Participants receive either the study drug NNZ-2591 or a matching placebo, administered orally twice daily.
Visits occur throughout the treatment period to monitor efficacy and safety.
Duration - 2 weeks
Participants undergo a safety follow-up period immediately after completing treatment to monitor for any adverse effects.
Visits focus on safety assessments following treatment completion.
Trial Site Locations
Total: 7 locations
1
Neuren PMS-301 Site#111
San Rafael, California, United States, 94903
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2
Neuren PMS-301 Site#102
Chicago, Illinois, United States, 60612
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3
Neuren PMS-301 Site#109
Chevy Chase, Maryland, United States, 20815
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4
Neuren PMS-301 Site#106
Brookline, Massachusetts, United States, 02445
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5
Neuren PMS-301 Site#104
Lexington, Massachusetts, United States, 02421
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6
Neuren PMS-301 Site#101
New York, New York, United States, 10029
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7
Neuren PMS-301 Site#108
Cincinnati, Ohio, United States, 45229
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Research Team
M
Medical Information Lead
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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