A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: NNZ-2591, Placebo.
- Who it may be relevant to
- Registry conditions: Phelan-McDermid Syndrome. Basic parameters: 3 years — 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Canada
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome
Overview
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.
Detailed description
After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome ages 3-12 years of age will enter the 4-week Screening Period and undergo assessments for eligibility, baseline characteristics and symptom severity.
Once eligibility is confirmed, participants will be randomized in a 1:1 ratio to receive either orally administered NNZ-2591 or matching placebo during the 13-week Treatment Period. Subsequently, a 2-week safety follow-up period will occur immediately after the completion of the Treatment Period.
Interventions
- Drug NNZ-2591
The study drug will be administered twice daily orally. - Drug Placebo
The study drug will be administered twice daily orally.
Primary outcome measures
- 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]
- 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. [Time frame: Week 13]
Secondary outcome measures (6)
- Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score. [Time frame: Week 13]
- Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. [Time frame: Week 13]
- Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores. [Time frame: Week 13]
- Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores. [Time frame: Week 13]
- Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMSA-S overall score. [Time frame: Week 13]
- Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores. [Time frame: Week 13]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 11 centers
- Neuren PMS-301 Site#122 — Glendale
- Neuren PMS-301 Site #112 — Palo Alto
- Neuren PMS-301 Site#103 — San Diego
- Neuren PMS-301 Site#111 — San Rafael
- Neuren PMS-301 Site#102 — Chicago
- Neuren PMS-301 Site#109 — Chevy Chase
- Neuren PMS-301 Site#106 — Brookline
- Neuren PMS-301 Site#104 — Lexington
- … and 3 more centers
Canada · 1 center
- Neuren PMS-301 Site#201 — Toronto
Identifiers
NCT: NCT07281079 · NEU-2591-PMS-301