NCT07846215尚未招募2 期
A Phase II, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of Basmisanil in Participants Aged 2 to 14 Years Old With Dup15q Syndrome
Newleos Therapeutics, Inc.1 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2026年12月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Change in quantitative electroencephalogram (qEEG) beta-band power
研究概览
简要总结
The primary goal of this Phase 2 clinical trial is to determine the effects of a selective negative allosteric modulator (NAM) of GABAA α5 receptor, Basmisanil (NTX-1511), on brain wave (EEG) characteristics in children and adolescents with Dup15q syndrome. The main questions this trial aims to answer are:
- How does NTX-1511 affect brain waves (as measured by EEG) in subjects with Dup15q syndrome?
- Does NTX-1511 impact other developmental features of subjects with Dup15q syndrome?
Researchers will compare the effects of NTX-1511 with matching placebo (look-alike granules that contain no drug).
Participants will:
- Take NTX-1511 or matching placebo 3 times every day for 12 weeks
- Visit the clinic 7 times over the course of 23 weeks
研究设计
- 研究类型
- 干预性
- 分配方式
- 随机
- 干预模型
- 平行分组
- 主要目的
- 治疗
- 盲法
- 三盲 (受试者、医护人员、研究者)
入排标准
- 年龄范围
- 2 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- Parent, caregiver, or legally authorized representatives has provided written informed consent for the study and is willing to comply with all requirements of the protocol
- Male or female aged 2 to 14 years inclusive at time of informed consent signature
- Documented duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2.-q13.1 (BP2-BP3) segment.
- Dup15q syndrome Clinician Global Impression of Severity Scale (Dup15q CGI-S) overall severity score ≥ 3 (at least mildly impaired)
- Key
排除标准
- Pathogenic or likely pathogenic genomic variant outside of the BP2-BP3 segment. Individuals with an isodicentric 15q chromosome and a history of epilepsy
- Uncontrolled epilepsy (in individuals with interstitial BP2-BP3 duplication or triplication) indicated by: - Use of rescue medication(s) to treat more than one seizure episode or seizure cluster per month, on average in the past 6 months, or -Concomitant chronic use of more than four anti-epileptic medications, or -Status epilepticus within the past 6 months requiring hospitalization for treatment of the status epilepticus, or -Any implanted devices to treat drug-resistant epilepsy
- Changes to medications within 6-8 weeks prior to screening
- Changes to non-pharmacological/behavioral therapies within 6 weeks prior to screening
研究组 & 干预措施
Placebo Comparator: Placebo
Placebo Comparator
干预措施: Placebo (Drug)
Experimental: NTX-1511
Active Comparator
干预措施: Basmisanil (Drug)
结局指标
主要结局
Change in quantitative electroencephalogram (qEEG) beta-band power
时间窗: Baseline to Week 12
次要结局
- Incidence and severity of adverse events (AEs) and severe AEs (SAEs)(Baseline to Week 12)
- Incidence of treatment discontinuations due to Adverse Events(Baseline to Week 12)
- Incidence of abnormalities in vital signs, electrocardiograms (ECGs) (PR, QRS, and QT intervals, as well as any other dysrhythmias or abnormal wave), laboratory results (chemistry, hematology and urinalysis), and/or physical examination(Baseline to Week 12)
- Change in Dup15q Clinical global impression of severity (CGI-s) and change (CGI-C)(Baseline to Week 12)
- Change in Vineland Adaptive Behavior Scales -Third Edition (Vineland-3) subdomain Growth Scale Value (GSV) scores(Baseline to Week 12)
- Change in Behavior Assessment System for Children - Third Edition (BASC-3) composite and scale scores(Baseline to Week 12)
- Change in Aberrant Behavior Checklist - Second Edition Community Version (ABC-2-C) domain scores(Baseline to Week 12)
- Change from baseline in frequency, duration, and type of any seizures as reported in a seizure diary by caregivers(Baseline to Week 12)
- Treatment-emergent abnormal changes in electroencephalograms(Baseline to Week 12)
研究者
研究点 (1)
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标识符
- NCT 编号
- NCT07846215
- 其他研究编号
- NTX-1511-201
日期
- 首次提交
- (12天前)
- 首次发布
- (昨天)
- 主要完成日期
- (2年后)
- 研究完成日期
- (2年后)
- 最近核实
- (29天前)
- 最近更新
- (昨天)
监管与共享
- FDA 监管药物
- 是
- FDA 监管器械
- 否
- 个体参与者数据共享计划
- 否
- 是否有结果
- 否
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