D - ND

FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE0 个研究点目标入组 14 人开始时间: 2020年11月4日最近更新: 2020年11月9日

适应症

试验速览

暂无简介。

•Informed Consent signed, before any trial procedures are performed
•Male or female patients
•Age =18 years
•Confirmed diagnosis of Fabry disease and concomitant treatment with standard dose of agalsidase alfa for at least 1 years, without any dose modification
•Presence of continuous gastrointestinal involvement for the previous 4 months before the screening, with at least 1 symptom reported in the GRSG questionnaire
•Elevated plasma lyso-GL3 (plasma lyso-Gl3 > 7 ng/ml)
•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 7
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 7

•plasma lyso-Gl3= 7 ng/ml at baseline and/or no evidences of GI symptoms.
•Patients with a diagnosis of chronic inflammatory diseases defined according to the criteria of the International Classification: gastroenterological, rheumatologic, immunologic autoimmune diseases
•Patients diagnosed with any type of cancer in evolution
•Patients diagnosed with infectious disease certified through microbiological tests performed in the last 12 months (included in the medical history of the patient)
•Patients with IBD, Celiac disease or other inflammatory chronic disease such as Crohn disease or rectal ulcerative colitis (RUC )
•Pregnancy or lactation
•Concomitant medication with chloroquine, amiodarone, benoquin or gentamycin, due to a theoretical risk of inhibition of intra-cellular a- Gal A activity.
•Known allergy to one or more of the components of agalsidase beta

发起方

FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE