Treatment with HMG-COA reductase inhibitor (simvastatin) of growth and bone abnormalities in children with Noonan syndrome : a phase III randomised, double-blind, placebo-controlled therapeutic trial - RASTAT

niversity Hospital Toulouse0 个研究点目标入组 62 人开始时间: 2016年3月30日最近更新: 2018年9月10日

试验速览

暂无简介。

•- Genetically confirmed Noonan syndrome (mutations in PTPN11, SOS1, RAF1, KRAS, RIT1, SPRED, or SHOC2 genes)
•- Female child between 6 to 15 years, without menses, and with bone age = 13 years
•or male child between 6 to 16 years, and with bone age = 14 years
•- Decreased growth velocity (= -1 SDS) and/or short stature (height = - 2 SDS or = -1.5 SDS under target height)
•- Informed consent obtained from child and parents
•- Participants with social security insurance or equivalent social protection.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 62
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•- Weight < 20 kg
•- Contraindication to simvastatin treatment:
•oProgressive liver disease, increased serum levels of alanine aminotransferase (> 1.5 upper limit of normal [ULN]), aspartate aminotransferase (> 1.5 ULN), or creatine phosphokinase (> 1.5 ULN)
•oKnown hypersensitivity to simvastatin
•oTreatment with CYP3A4 inhibitors (erythromycin, clarithromycin, ketoconazole, itraconazole cyclosporine, or danazol), gemfibrozil
•- Growth promoting therapies such as recombinant human GH or IGF-1 treatment during the last 3 months

发起方

niversity Hospital Toulouse