Pharmacological Treatment of Rett Syndrome With 3-Hydroxy-3 Methylglutaryl-coenzyme A Reductase Inhibitor-Lovastatin (Mevacor)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Gait Velocity as Measured by GAITRite System
研究概览
简要总结
This is a phase 2 , open label, dose escalating study of Lovastatin in Rett syndrome.
详细描述
Excess neuronal cholesterol plays a role in the pathophysiology of Rett syndrome(RTT) and the investigators hypothesise that inhibition of cholesterol synthesis in the CNS will reduce neuronal cholesterol and lead to improvement in Rett syndrome related symptoms.
Goal: To determine the optimal dosing regiment of Lovastatin for patients with RTT and to identify the most appropriate primary outcome measure for the subsequent Phase 3 study.
Phase 2, dose escalation study.
Primary outcome: Gait speed Secondary outcomes: respiratory function, cognition, EEG (encephalopathy), Rett syndrome severity scale, neurodevelopmental assessment (QOL)
20 ambulatory female patients with genetically confirmed.
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 —（Child, Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Females patients,
- •Genetically confirmed RTT,
- •Ambulatory.
排除标准
- •Presence of co morbid non-Rett related disease,
- •History of adverse reaction/hypersensitivity to statins,
- •Levels of aspartate-amino trans-ferase (AST), alanine-amino transferase (ALT) or creatine kinase (CK) triple the normal values,
- •Active liver disease,
- •Concomitant use of strong CYP3A4 inhibitors,
- •Condition predis-posing to renal failure secondary to rhabdomyolysis (scoliosis surgery within 6 months),
- •Oral contraceptives use.
研究组 & 干预措施
Open label
Treatment with Lovastatin, dose escalating trial according to the following schedule:
10 mg daily for 8 week 20 mg daily for 8 weeks 40 mg daily for 16 weeks.
干预措施: Lovastatin (Drug)
结局指标
主要结局
Gait Velocity as Measured by GAITRite System
时间窗: During final week of treatment, week 32
To perform quantitative gait assessment using a computerized walkway with embedded pressure sendors (GAIT rite)
次要结局
- Visual Memory Novelty Score as Assessed by TobiiTX 300 Eyetracking System(final week of treatment, Week 32)
研究者
Aleksandra Djukic
Professor of Clinical Neurology, Director, Tri State Rett Syndrome Center
Montefiore Medical Center