Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07281079
The ClinicalTrials.gov registry entry for NCT07281079 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 160 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 72-participant average among 4 other Phelan-McDermid Syndrome trials with a reported enrollment target (122% higher). The listed sponsor is Neuren Pharmaceuticals Limited, which has 10 total studies on file at ClinicalTrials.gov.
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 2 study locations spanning 2 distinct geographic areas - top geographies include 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.
Learn More About Clinical Trials
Similar trials for Phelan-McDermid Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
RECRUITING · Phase 1
-
Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
ACTIVE NOT RECRUITING
-
Mitochondrial Dysfunction in Phelan-McDermid Syndrome
COMPLETED
-
Mapping the Phenotype in Adults With Phelan-McDermid Syndrome
COMPLETED
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.