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NCT07281079 · ClinicalTrials.gov registry record · Phase 3
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
A Phase 3 study of Phelan-McDermid Syndrome, sponsored by Neuren Pharmaceuticals Limited.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 160
- Enrollment target
- 2
- Study locations
NCT07281079: Recruiting Phase 3 study of Phelan-McDermid Syndrome, sponsored by Neuren Pharmaceuticals Limited.
NCT07281079 is a Phase 3 study of Phelan-McDermid Syndrome that is actively recruiting participants, run by Neuren Pharmaceuticals Limited. The registered enrollment target is 160 participants, above the 72-participant average among 4 other Phelan-McDermid Syndrome trials with a reported enrollment target (122% higher). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07281079, a Phase 3 study of Phelan-McDermid Syndrome, is actively recruiting participants, sponsored by Neuren Pharmaceuticals Limited.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 160 participants
- Enrollment target
- 2
- Study locations
Study 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.
Primary Outcome
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.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG NNZ-2591
Study Locations (2)
California
- Neuren PMS-301 Site#111 - San Rafael
Maryland
- Neuren PMS-301 Site#109 - Chevy Chase
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 160 participants |
| Start Date | 2025-11-12 |
| Est. Completion | 2027-11-15 |
| Phase | Phase 3 |
What NCT07281079 shows while recruiting
NCT07281079 is an interventional study that assigns participants to a tested intervention. The registered 160 participants enrollment target is mid-sized for trials with a published cap, above the 72-participant average among 4 other Phelan-McDermid Syndrome trials with a reported enrollment target (122% higher).
The record links to 1 condition, with Phelan-McDermid Syndrome appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT07281079 reports a single indexed study location in California, Maryland.
Frequently Asked Questions
What is clinical trial NCT07281079 about?
NCT07281079 is a clinical study titled "A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome". 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.
What is the current status of trial NCT07281079?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 160 participants. The study started on 2025-11-12. Estimated completion is 2027-11-15.
What conditions does trial NCT07281079 study?
This clinical trial studies the following conditions: Phelan-McDermid Syndrome.
What interventions are being tested in trial NCT07281079?
The interventions under investigation include: Placebo (DRUG), NNZ-2591 (DRUG).
Who is sponsoring clinical trial NCT07281079?
This trial is sponsored by Neuren Pharmaceuticals Limited, which has 10 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07281079 being conducted?
This trial has 2 study locations across California, Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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