NCT00731016已完成2 期
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
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
- 15
- 试验地点
- 1
- 主要终点
- To evaluate the tolerance and efficacy of pravastatin and zoledronic acid in combination on the patient's weight, height and bone metabolism in Progeria treatment
研究概览
简要总结
We suggest treating the Hutchinson-Gilford Progeria Syndrome by two molecules (zoledronic acid and pravastatin).The therapeutic approach which we propose has for objectives to reduce, to prevent or to delay the gravest infringements of the disease, to prolong the life of the children, and in a more general way, aim at improving their living conditions.
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
- All
- 接受健康志愿者
- 否
入选标准
- •Molecularly characterised patients with a known mutation of their LMNA gene leading to the production of a farnesylated prelamin A, whether truncated or not
- •Patients must be able to travel and consult in Marseille, France for necessary explorations planned at the inclusion step, then following the protocol flow
- •chart for zoledronic acid injections and follow-up visits
- •Patient older than 3 years
- •Patients affiliated or beneficiary of a legal medical insurance
- •Adult patients certifying they have been properly informed about the protocol, and they signed a written consent form. Children and/or disabled patients whose parents/legal tutor have been informed and have signed a written consent form
排除标准
- •Known hypersensitivity to pravastatin or zoledronic acid
- •Seric transaminase levels higher than 3 times of normal value
- •CPK level higher than 5 times of normal value
- •Creatininemia higher than 0.5mg/dl or 44mM, or creatinin clearance lower than 70ml/min/1.73m3
- •Presence of dental troubles, or recent dental trouble
- •Maxillary osteonecrosis or bone nakedness antecedent
- •Congenital galacosemia, glucose or galactose maladsorption syndrome, lactase deficiency
- •Every other pathology thought to be incompatible with proposed treatment by the investigator
- •Under treatment that can interfere with pravastatin and/or zoledronate metabolisms
研究组 & 干预措施
1
Other
Zoledronic acid, pravastatin
干预措施: Zoledronic acid, pravastatin (Drug)
结局指标
主要结局
To evaluate the tolerance and efficacy of pravastatin and zoledronic acid in combination on the patient's weight, height and bone metabolism in Progeria treatment
时间窗: 48 months
次要结局
- To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms(48 months)
研究者
研究点 (1)
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