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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 180
- 试验地点
- 9
- 主要终点
- Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
NNZ-2591 Arm
The total duration of this study for each participant will be up to up to 56 weeks.
干预措施: NNZ-2591 (Drug)
结局指标
主要结局
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
时间窗: Baseline through Safety Follow-Up (Month 12)
Change from Baseline PR Interval (ms QRS interval (ms)
Long-term safety and tolerability of NNZ-2591 as assessed by the incidence of adverse events across participants
时间窗: Baseline through Safety Follow-Up (Month 12)
Incidence of TEAEs, AESI and SAEs across participants
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of ECG parameters events across participants.
时间窗: Baseline through Month 12
Change from Baseline in ECG Heart Rate (bpm)
Long-term safety and tolerability of NNZ-2591 as assessed by changes from Baseline assessments of vital sign parameters events across participants.
时间窗: Baseline through Month 12
Change from Baseline for heart rate (bpm)
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant clinical laboratory parameters events across participants.
时间窗: Baseline through Month 12
Incidence of abnormal and clinically significant laboratory parameters
Long-term safety and tolerability of NNZ-2591 as incidence of abnormal, clinically significant physical examination findings across participants.
时间窗: Baseline through Month 12
Incidence of abnormal, clinically significant physical examination findings
次要结局
- Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score(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.(Months 3 and 12)
- Efficacy of NNZ-2591 as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores.(Months 3 and 12)
- Efficacy of NNZ-2591 as measured by the Caregiver Impression of Change (CIC) domain scores.(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.(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.(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.(Months 3 and 12)