Actively Recruiting
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Led by Neuren Pharmaceuticals Limited · Updated on 2026-05-08
160
Participants Needed
5
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 aged 3 to 12 years at the time of informed consent
- Clinical diagnosis of Phelan-McDermid syndrome with documented disease-causing SHANK3 genetic abnormality
- Body weight of at least 10 kg at screening
- Phelan-McDermid Syndrome Assessment of Change (PMSA-S) overall score of 3 or higher at screening and baseline visits
- No active regression or loss of skills
You will not qualify if you...
- Use of exclusionary or unstable medication regimens as defined by the protocol
- Current treatment with more than 3 allowable psychotropic medications
- Participants with seizures controlled on 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 1 seizure in the past 12 months (except a single febrile seizure within 6 months without rescue medication)
- Abnormal liver function test results during screening
- Abnormal QT interval on screening ECG as defined by the protocol
AI-Screening
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Trial Site Locations
Total: 5 locations
1
Neuren PMS-301 Site#111
San Rafael, California, United States, 94903
Actively Recruiting
2
Neuren PMS-301 Site#109
Chevy Chase, Maryland, United States, 20815
Actively Recruiting
3
Neuren PMS-301 Site#106
Brookline, Massachusetts, United States, 02445
Actively Recruiting
4
Neuren PMS-301 Site#101
New York, New York, United States, 10029
Actively Recruiting
5
Neuren PMS-301 Site#108
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
M
Medical Information Lead
CONTACT
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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