NCT05011851已完成2 期
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 17
- 试验地点
- 3
- 主要终点
- Safety and Tolerability
研究概览
简要总结
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Angelman syndrome
详细描述
The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution, 50mg/L, in children and adolescents with Angelman syndrome. The secondary purpose is to investigate measures of efficacy of subjects will receive treatment of 50mg/mL orally administered NNZ-2591 for a total of 13 weeks
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 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of AS with a documented disease-causing genetic etiology known to impact maternally derived UBE3A expression in brain.
- •Males or females aged 3-17 years
- •Body Weight of >12Kg
- •Subjects with a Clinical Global Impression - Severity (CGI-S) score of 3 or greater
- •Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
- •Each subject must be able to swallow the study medication provided as a liquid solution.
- •Caregiver(s) must have sufficient English language skills.
排除标准
- •Mosaicism for disease-causing mutation.
- •Clinically Significant abnormalities in safety laboratory testing or vital signs at screening
- •Abnormal QTcF interval or prolongation at Screening.
- •Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.
- •Unstable or changes to Psychotropic treatment 2 weeks prior to screening .
- •Excluded concomitant treatments
- •Actively undergoing regression or loss of skills.
- •Unstable seizure profile.
- •Current clinically significant renal conditions and abnormalities
- •Current clinically significant cardiovascular, hepatic, gastrointestinal, respiratory, endocrine disease, or clinically significant organ impairment.
- •Current clinically significant hypo or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes.
- •Has planned surgery during the study.
- •History of, or current, cerebrovascular disease or brain trauma.
- •History of, or current catatonia or catatonia-like symptoms.
- •History of, or current, malignancy.
- •Current major or persistent depressive disorder (including bipolar depression).
- •Significant, uncorrected visual or uncorrected hearing impairment.
- •Allergy to strawberry.
- •Positive pregnancy test
- •Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study
研究组 & 干预措施
NNZ-2591
Experimental
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
干预措施: NNZ-2591 (Drug)
结局指标
主要结局
Safety and Tolerability
时间窗: 13 weeks
To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591.
Pharmacokinetic - Typical AUC24 of 30kg Child
时间窗: 13 weeks
Area under the concentration-time curve of NNZ-2591 over 24 hours
Pharmacokinetic - Typical t1/2 in 30 kg Child
时间窗: 13 weeks
Apparent terminal elimination half-life of NNZ-2591
次要结局
- Angelman syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I)(13 weeks)
- Caregiver Impression of Improvement : Overall Score(13 weeks)
- Angelman syndrome-specific Clinical Global Impression Scale - Severity (CGI-S): Overall Score(13 weeks)
- Angelman syndrome Clinician Domain Specific Rating Scale (AS-DSRS)(13 weeks)
- Caregiver Top 3 Concerns(13 weeks)
- MacArthur-Bates Communicative Development Inventory (MB-CDI)(13 weeks)
- Observer-Reported Communication Ability (ORCA)(13 weeks)
- Aberrant Behavior Checklist-2 (ABC-2)(13 weeks)
- Child Sleep Habits Questionnaire (CSHQ)(13 weeks)
- Gastrointestinal Health Questionnaire (GIHQ)(13 weeks)
- Vineland Adaptive Behavior Scales-3, Interview version(13 weeks)
- Exploratory efficacy measurement(13 weeks)
- Quality of Life Inventory-Disability (QI-Disability)(13 weeks)
- Impact of Childhood Neurological Disability (ICND)(13 weeks)
研究者
研究点 (3)
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