An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат NNZ-2591
  The study drug will be administered twice daily orally.

Первичные конечные точки

• Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: Baseline through Month 12]

Вторичные конечные точки (7)

• Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score [Срок оценки: 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. [Срок оценки: Months 3 and 12]
• Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores. [Срок оценки: Months 3 and 12]
• Efficacy of NNZ-2591 as measured by the Caregiver Impression of Change (CIC) domain scores. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: 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. [Срок оценки: Months 3 and 12]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 2 центра

• Neuren PMS-302 Site#111 — San Rafael
• Neuren PMS-302 Site#109 — Chevy Chase

Идентификаторы

NCT: NCT07593391 · NEU-2591-PMS-302

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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