Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con CalcifediolMulticentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathy

Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau0 个研究点开始时间: 2008年1月25日最近更新: 2012年3月19日

适应症

试验速览

暂无简介。

•1. Men and women of age compressed between 18 and 60 years old
•2. Demonstartion of the state of carrier of a mutation in the dysferlin gene and demostration of reduction of dysferlin protein expression in monocytes
•2. Symptomatic carriers of a mutation in the dysferlin gene demonstrated by any of the following:
•a. Hyperckemia
•b. Weakness
•c. Modification of the normal structure of gastrocnemius demonstrated by magnetic resonance imaging
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•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

•1. Patients currently treated with calcidefiol
•2. Pregnancy
•3. Patients affected of renal failure
•4. Patients affected of hypercalcemia
•5. Patients who cannot follow the physical tests of the study
•6. Patients with allergy to calcifediol
•7. Patients who are or have been included in any other Medical study within a minimal period of 30 days

发起方