Improvement of Synaptic Plasticity and Cognitive Function in RAS Pathway Disorders
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Long-term potentiation (LTP)-like plasticity measured with transcranial magnetic stimulation (TMS)
研究概览
简要总结
The project is targeting cognitive impairment, one of the main health problems of patients with RAS pathway disorders. The aim of this study is to translate findings of animal studies to humans. This has been done by the applicants successfully for Lovastatin in Nf1. This result will be transferred to patients with Noonan Syndrome. lamotrigine is most likely a more effective and promising substance improving synaptic plasticity and consecutive cognitive function. It is expected that both substances are improving synaptic plasticity as well as alertness and changes in alertness may be a precondition for improvement of cognition.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Group 1: NS, Group 2: NF1 (both genetically assured)
- •Age ≥16 years
- •The adolescent (≥16) and legal guardian who are capable to give their consent and understand the aim and rationale of the study. In case of doubts, an independent medical practitioner will evaluate the capacity to consent.
- •Signed informed consent if ≥ 16 years and legal guardian.
- •Persons who are ≥ 18 years old and capable to give their consent and understand the aim and rationale of the study. In case of doubts, an independent medical practitioner will evaluate the capacity to consent.
- •Signed informed consent if ≥ 18 years.
- •Male participants and female participants who are not capable of bearing children or who use a method of contraception that is medically approved by the health authority of the respective country.
排除标准
- •Medication with known CNS effects
- •Severe mental retardation
- •Side effects during previous medication with and contraindications for LTG and/or LOV and/or TMS
- •Psychiatric diseases
- •Previous history of allergic reactions with LTG and LOV medications
- •Potentially unreliable patients
- •Patients who are not suitable for the study in the opinion of the investigator
- •Pregnancy (incl. positive urine pregnancy test)
- •Persons who are incapable of giving consent or do not understand the aim or rationale of the study.
研究组 & 干预措施
Exp. I: Noonan Syndrome - Lovastatin
200 mg Lovastatin daily for four days / Lovastatin-placebo (cross-over) prior to transcranial magnetic stimulation and test of attentional performance
干预措施: Lovastatin (Drug)
Exp. II: Noonan Syndrome - Lamotrigine
300 mg Lamotrigine single dose / Lamotrigine-placebo prior to transcranial magnetic stimulation and test of attentional performance
干预措施: Lamotrigine (Drug)
Exp. III: Neurofibromatosis Type 1 - Lamotrigine
300 mg Lamotrigine single dose / Lamotrigine-placebo prior to transcranial magnetic stimulation and test of attentional performance
干预措施: Lamotrigine (Drug)
结局指标
主要结局
Long-term potentiation (LTP)-like plasticity measured with transcranial magnetic stimulation (TMS)
时间窗: 12 months
Changes in peak-to-peak amplitudes of motor evoked potentials (MEP)
次要结局
- Difference between the neuropsychological testing of attention (Test of attentional performance) after placebo and after medication (LTG and LOV)(12 months)
- Differences in short interval cortical inhibition (SICI) after placebo and after medication (LTG and LOV)(12 months)