A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Neuren Pharmaceuticals Limited · NCT07281079
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 3–12 · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 3
- Target enrollment
- 160
- US sites recruiting
- 17
- Study start
- November 12, 2025
- Record last updated
- September 28, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study 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.
Being studied: NNZ-2591; Placebo
Conditions: Phelan-McDermid Syndrome
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full criteria
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.
- 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.
Recruiting US locations
Neuren PMS-301 Site #116
Birmingham, AL 35233
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#122
Glendale, CA 91203
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site #112
Palo Alto, CA 94305
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#103
San Diego, CA 92123
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#111
San Rafael, CA 94903
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site #107
Washington D.C., DC 20010
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site #105
Atlanta, GA 30322
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#102
Chicago, IL 60612
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#109
Chevy Chase, MD 20815
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#106
Brookline, MA 02445
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#104
Lexington, MA 02421
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site #117
Ann Arbor, MI 48109
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#101
New York, NY 10029
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#108
Cincinnati, OH 45229
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#110
Columbus, OH 43205
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site#115
Houston, TX 77030
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Neuren PMS-301 Site #113
Charlottesville, VA 22903
Site contact: medicalinformation@neurenpharma.com · 231-203-8050 · medicalinformation@neurenpharma.com
Study contacts (from the official record)
- Medical Information Lead · 231-203-8050 · medicalinformation@neurenpharma.com