A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Part of paid clinical trials in Glendale, California.

Sponsor
Neuren Pharmaceuticals Limited
Study ID
NCT07281079
Phase
PHASE3
Status
Recruiting
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Conditions

  • Phelan-McDermid Syndrome

Eligibility Criteria

Sex
ALL
Age
3 Years - 12 Years
Healthy Volunteers
Not accepted

Interventions

  • NNZ-2591 — DRUG
    The study drug will be administered twice daily orally.
  • Placebo — DRUG
    The study drug will be administered twice daily orally.

Study Details

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.

Key Dates

Start date
Nov 12, 2025
Status verified
Jun 2026
Primary completion
Oct 31, 2027
Completion
Nov 15, 2027

Study Design

Enrollment
160 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: NNZ-2591 Arm
    The total duration of this study for each participant will be up to approximately 17 to 19 weeks. Participants will be randomized in a 1:1 ratio to receive orally administered NNZ-2591 during the 13-week Treatment Period.
  • Placebo Comparator: Placebo Arm
    The total duration of this study for each participant will be up to approximately 17 to 19 weeks. Participants will be randomized in a 1:1 ratio to receive orally administered placebo matching NNZ-2591 during the 13-week Treatment Period.

Primary Outcome Measure

Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. [ Time Frame: Week 13 ]

Central Contacts

Locations (9)

FacilityCityStateZIPSite coordinators
Neuren PMS-301 Site#122GlendaleCalifornia91203
Neuren PMS-301 Site#103San DiegoCalifornia92123
Neuren PMS-301 Site#111San RafaelCalifornia94903
Neuren PMS-301 Site#102ChicagoIllinois60612
Neuren PMS-301 Site#109Chevy ChaseMaryland20815
Neuren PMS-301 Site#106BrooklineMassachusetts02445
Neuren PMS-301 Site#104LexingtonMassachusetts02421
Neuren PMS-301 Site#101New YorkNew York10029
Neuren PMS-301 Site#108CincinnatiOhio45229

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Neuren PMS-301· Glendale, CANeuren PMS-301· San Diego, CANeuren PMS-301· San Rafael, CANeuren PMS-301· Chicago, ILNeuren PMS-301· Chevy Chase, MDNeuren PMS-301· Brookline, MA

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