Rapamycin (Sirolimus) treatment for amyotrophic lateral sclerosis - RAP.ALS

AZIENDA OSPEDALIERO-UNIVERSITARIA POLICLINICO DI MODENA0 个研究点目标入组 63 人开始时间: 2018年1月23日最近更新: 2018年1月29日

适应症

试验速览

暂无简介。

•-Patient diagnosed with a laboratory supported , clinically probable” or definite” amyotrophic lateral sclerosis according to the Revised El Escorial criteria (Brooks, 2000)
•-Familial or sporadic ALS
•-Female or male patients aged between 18 and 75 years old
•-Disease duration from symptoms onset no longer than 18 months at the screening visit
•-Patient treated with a stable dose of Riluzole (100 mg/day) for at least 30 days prior to screening
•-Patients with a weight > 50 kg and a BMI =18
•-Patient with a FVC = 70 % predicted normal value for gender, height, and age at the screening visit
•-Patient able and willing to comply with study procedures as per protocol
•-Patient able to understand, and capable of providing informed consent at screening visit prior to any protocol-specific procedures
•-Use of effective contraception both for males and females
•Are the trial subjects under 18? no
•Number of subjects for this age range: 1
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 45
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 18

•-Prior use of Sirolimus
•-Prior allergy/sensitivity to Sirolimus or macrolides
•-Any medical disorder that would make immunosuppression contraindicated, including but not limited to, acute infections requiring antibiotics, patients with known diagnosis of HIV, TBC, hepatitis B or C infection or history of malignancy
•-Severe comorbidities (heart, renal, liver failure), autoimmune diseases or any type of interstitial lung disease
•-White blood cells<4,000/mm³, platelets count<100,000/mm³, hematocrit<30%
•-Patient who underwent non invasive ventilation, tracheotomy and /or gastrostomy
•-Women who are pregnant or breastfeeding
•-Participation in pharmacological studies within the last 30 days before screening
•-Patients with known SOD1 mutation

发起方

AZIENDA OSPEDALIERO-UNIVERSITARIA POLICLINICO DI MODENA