Recruiting NCT07593391
An Open-label 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.
- 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
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase 3 Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Orally Administered NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Overview
This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 in pediatric participants with Phelan- McDermid Syndrome.
Detailed description
After providing informed consent/assent, pediatric participants with Phelan-McDermid syndrome who participated in previous studies (NEU-2591-PMS-301 and NEU-2591-PMS-001) will undergo assessments for eligibility, baseline characteristics and symptom severity. Once eligibility is confirmed, participants will receive orally administered NNZ-2591 during the 52-week Treatment Period. 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.
Primary outcome measures
- Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants [Time frame: Baseline through Safety Follow-Up (Month 12)]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [Time frame: Baseline through Month 12]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [Time frame: Baseline through Safety Follow-Up (Month 12)]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [Time frame: Baseline through Safety Follow-Up (Month 12)]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [Time frame: Baseline through Safety Follow-Up (Month 12)]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [Time frame: Baseline through Safety Follow-Up (Month 12)]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants. [Time frame: Baseline through Safety Follow-Up (Month 12)]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [Time frame: Baseline through Month 12]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [Time frame: Baseline through Month 12]
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants. [Time frame: Baseline through Month 12]
Secondary outcome measures (7)
- Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score [Time frame: Months 3 and 12]
- Efficacy of NNZ-2591 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: Months 3 and 12]
- Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. [Time frame: Months 3 and 12]
- Efficacy of NNZ-2591 as measured by the Caregiver Impression of Change (CIC) domain scores. [Time frame: Months 3 and 12]
- Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) domain scores. [Time frame: Months 3 and 12]
- Efficacy of NNZ-2591 as measured by the change from baseline in Phelan-McDermid Syndrome Assessment of Severity (PMSA-S) overall score. [Time frame: Months 3 and 12]
- Efficacy of NNZ-2591 as measured by the change from baseline in PMS Clinician Domain Specific Rating Scale (PMS-DSRS) scores. [Time frame: Months 3 and 12]
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 for the antecedent study.
- Participant must have completed all applicable study visits for the antecedent study in which they participated.
- Body weight ≥ 10 kg at Screening/Baseline.
- 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.
- 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
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Neuren PMS-302 Site#111 — San Rafael
- Neuren PMS-302 Site#109 — Chevy Chase
Identifiers
NCT: NCT07593391 · NEU-2591-PMS-302