An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 20
- 试验地点
- 4
- 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 Prader-Willi 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 in children and adolescents with Prader-Willi Syndrome. The secondary purpose is to investigate measures of efficacy. Subjects will receive treatment with NNZ-2591 oral solution (50 mg/mL) doses 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
- 4 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of PWS with a documented disease-causing genetic abnormality of the chromosome 15q11-q13 confirmed by DNA methylation and microarray.
- •Males or females aged 4-12 years, inclusive.
- •Body weight of 12 kg to 100kg (inclusive) at Baseline.
- •Subjects with a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at the Screening visit.
- •Must currently be on treatment with growth hormone.
- •Each subject must be able to swallow the study medication provided as a liquid solution.
- •Caregiver(s) must have sufficient English language skills.
- •Subject and caregiver must reside in the US and have been resident in the US for at least 3 months prior to screening.
排除标准
- •Body weight <12 kg or >100 kg at Baseline.
- •HbA1c values above 7% at the Screening visit.
- •Clinically significant abnormalities in safety laboratory tests and vital signs at Screening.
- •Positive pregnancy test at the Screening visit.
- •Positive drugs of abuse screen not explained by concomitant medications.
- •Abnormal QTcF interval or prolongation at Screening.
- •Any other clinically significant finding on ECG at the Screening visit.
- •Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or Baseline.
- •Previous COVID 19 infection with last 12 months that required hospitalization.
- •Previous COVD-19 infection involving multi-organ systems, resulting in Multisystem Inflammatory Syndrome in Children (MIS-C) or with clinically significant long term effects.
- •COVID-19 infection associated with acute kidney injury (AKI) or renal conditions.
- •Renal conditions or abnormalities identified in laboratory testing, imaging or medical history.
- •Liver conditions and Hepatic abnormalities.
- •Vision abnormalities and Ocular conditions.
- •Excluded concomitant treatments.
- •Unstable seizure profile.
- •Current clinically significant cardiovascular, gastrointestinal, or respiratory disease, or clinically significant organ impairment, or endocrine disease with the exception of obesity and controlled hypothyroidism.
- •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 hearing impairment.
- •Allergy to strawberry.
- •Has participated in another interventional clinical study within 30 days prior to start of Screening.
- •Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study.
研究组 & 干预措施
NNZ-2591
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 - Measurement of Cmax
时间窗: 13 weeks
Maximum observed concentration (Cmax) of NNZ-2591
Pharmacokinetic - Measurement of AUC
时间窗: 13 weeks
Area under the concentration-time curve of NNZ-2591
Pharmacokinetic - Measurement of time to Cmax
时间窗: 13 weeks
Time to Cmax of NNZ-2591
Pharmacokinetic - Measurement of t1/2
时间窗: 13 weeks
Apparent terminal elimination half-life of NNZ-2591
次要结局
- Exploratory efficacy measurement(13 weeks)