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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 160
- 试验地点
- 17
- 主要终点
- Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) This is a double-blind study in which pediatric participants, caregivers, investigators, and the sponsor are blinded to study drug for the duration of study. Dose escalation, dose modification, and treatment discontinuation will be performed in a blinded manner.
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.
- •Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK
- •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.
排除标准
- •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.
研究组 & 干预措施
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.
干预措施: Placebo (Drug)
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.
干预措施: NNZ-2591 (Drug)
结局指标
主要结局
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score.
时间窗: Week 13
Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) overall score. The PMSA-C scores range from 1 to 7 with 1 indicating very much improved and 7 indicating very much worse.
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.
时间窗: 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. A higher raw score for the receptive communication subdomain indicates better adaptive behavior.
次要结局
- Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) overall score.(Week 13)
- Efficacy of NNZ-2591 compared with placebo as measured by the Phelan-McDermid Syndrome Assessment of Change (PMSA-C) domain scores.(Week 13)
- Efficacy of NNZ-2591 compared with placebo as measured by the Caregiver Impression of Change (CIC) domain scores.(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.(Week 13)
- Efficacy of NNZ-2591 compared with placebo as measured by the change from baseline in PMSA-S overall score.(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.(Week 13)