Study of NNZ-2591 for Phelan-McDermid Syndrome in Children
This study is testing a medication called NNZ-2591 for children with Phelan-McDermid Syndrome. We want to see if NNZ-2591 can help improve symptoms compared to a placebo (an inactive substance). Children aged 3 to 12 years with a confirmed diagnosis of Phelan-McDermid Syndrome and a specific genetic change (SHANK3 abnormality) can join. We will measure success by looking at changes in a special assessment for Phelan-McDermid Syndrome (PMSA-C) and improvements in communication skills (Vineland-3). The current status of this study is unclear, but it plans to enroll 160 participants.
- Study design
- This is a Phase 3, randomized, double-blind study, meaning participants will be randomly assigned to receive either NNZ-2591 or a placebo, and neither you nor the study team will know which you are receiving. It plans to enroll 160 participants.
- What's involved
- After an initial 4-week screening period, participants will take either NNZ-2591 or placebo twice daily orally for 13 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- There will be a 2-week safety follow-up period immediately after the 13-week treatment period.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
At a glance
Conditions
NCT07281079
Where you'd take part
This study runs at 15 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Neuren PMS-301 Site #112
Palo Alto, Californiastudy coordinator listed
Recruiting
Neuren PMS-301 Site #113
Charlottesville, Virginiastudy coordinator listed
Recruiting
Neuren PMS-301 Site #116
Birmingham, Alabamastudy coordinator listed
Recruiting
Neuren PMS-301 Site #117
Ann Arbor, Michiganstudy coordinator listed
Recruiting
Neuren PMS-301 Site#101
New York, New Yorkstudy coordinator listed
Recruiting
Neuren PMS-301 Site#102
Chicago, Illinoisstudy coordinator listed
Recruiting
Neuren PMS-301 Site#103
San Diego, Californiastudy coordinator listed
Recruiting
Neuren PMS-301 Site#104
Lexington, Massachusettsstudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. The PMSA-C scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.
- 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.Week 13
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. A higher raw score for the receptive communication subdomain indicates better adaptive behavior.