Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen. - FOR-DMD

AZIENDA OSPEDALIERA DI PADOVA0 个研究点目标入组 300 人开始时间: 2012年12月28日最近更新: 2013年8月5日

适应症

试验速览

暂无简介。

•Subjects with a confirmed diagnosis of DMD (defined as evidence of clinical signs of DMD and a confirmed mutation in the dystrophy gene (deletion, duplication or point mutation), age of at least 4 years and less than 8 years. ability to risde from the floor independently, ability and willingness of parents or legal tutor to give consent and to respect the scheduled appointments, ability to obtain consistent measures of FVC.
•Are the trial subjects under 18? no
•Number of subjects for this age range: 300
•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

•Significant renal or hepatic insufficiency; immunosuppression or other controindications to corticosteroid therapy; history of chronic systemic fungal or viral infections; diabetes mellitus; idiopatic hypercalciuria; luck of immunity to Varicella-Zoster virus and refusal of the vaccination; evidence of symptomatic cardiomyopathy; allergy/sensitivity to the study drugs or their formulations, current or previous treatment with corticosteroids or other immunosuppressive treatments for DMD or other signs of persistent inhability to swallow the tablets, severe behavioral problems, including autism, previous and current medical condition, medical history, physical findings or laboratory abnormalities that may compromise the safety or affect the evaluation of the results of the study; weight below 13 Kg; exposure to any investigational drug, currently or in the 3 months prior to starting thw study treatment.

发起方

AZIENDA OSPEDALIERA DI PADOVA