A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 82
- 试验地点
- 12
- 主要终点
- Adverse events
研究概览
简要总结
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett syndrome in children and adolescents.
详细描述
Rett syndrome is a neurodevelopmental disorder primarily affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of regression with onset of systemic and neurological signs. The CNS symptoms of Rett syndrome include learning disability, autism symptomatology and epilepsy and these can be severe and highly debilitating. Affected individuals also show signs of autonomic dysfunction, reflected in cardiovascular and respiratory abnormalities. There is no currently effective treatment for Rett syndrome.
This study will investigate the safety, tolerability and blood pharmacokinetics of treatment with oral administration of NNZ-2566 at 50 mg/kg, 100 mg/kg, 200 mg/kg BID, or placebo BID, in children and adolescent females with Rett syndrome. The study also will also investigate measures of efficacy and biomarkers during treatment.
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)
入排标准
- 年龄范围
- 5 Years 至 15 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene.
- •Age 5 - 15 years.
- •Weight at Screening and Baseline between 15.0 kg-100.0 kg (at least 15.0 kg and no greater than 100.0 kg).
- •Each subject must be able to swallow the study medication provided as a liquid solution, or via gastrostomy tube.
排除标准
- •Actively undergoing neurological regression
- •Abnormal QT interval, prolongation or significant cardiovascular history.
- •Current treatment with insulin.
- •Anti-convulsants with liver enzyme inducing effects.
- •Unstable seizure profile.
- •Excluded concomitant medications.
- •Current clinically significant (as determined by the investigator). cardiovascular, renal, hepatic, or respiratory disease.
- •Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication.
- •History of, or current cerebrovascular disease or brain trauma.
- •History of, or current clinically significant endocrine disorder, e.g. hypo- or hyperthyroidism, or diabetes mellitus.
- •History of, or current, malignancy.
- •Significant hearing and/or visual impairments that may affect ability to complete the test procedures.
- •Allergy to strawberry.
研究组 & 干预措施
NNZ-2566
Glycyl-L-2-Methylpropyl-L-Glutamic Acid
干预措施: NNZ-2566 (Drug)
Placebo (strawberry flavored solution)
Strawberry flavored solution and Water for Injection
干预措施: Placebo (Drug)
结局指标
主要结局
Adverse events
时间窗: Through study completion, an average of 11 weeks
Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the three NNZ-2566 doses and placebo. SAEs and AEs will be examined throughout the study.
次要结局
- Clinical Global Impression of Improvement (CGI-I)(Through study completion, an average of 11 weeks)
- Motor Behaviour Assessment Scale (MBA)(Through study completion, an average of 11 weeks)
- Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS)(Through study completion, an average of 11 weeks)