ISRCTN05147495已完成不适用

Rational use of substrate reduction therapy and enzyme replacement therapy in patients with type I Gaucher disease: an open-label single-centre follow up study

Aragon Institute of Health Sciences [Instituto Aragonés de Ciencias de la Salud] (Spain)0 个研究点目标入组 40 人开始时间: 2011年4月18日最近更新: 2015年1月13日

适应症

试验速览

暂无简介。

•1. Patients = 18 years. Diagnosis of type I GD confirmed by glucocerebrosidase assay or by the presence of mutations in the glucocerebrosidase gene
•2. Patients who have received enzyme replacement therapy for at least 3 years with the same dose for at least the last 6 months
•3. Stable clinical and biological disease for 2 years confirmed with at least two assessments including the one performed at baseline

•1. History of oculomotor palsy, ataxia or other manifestations usually associated with type 3 GD
•2. Splenectomy before 18 years due to massive splenomegaly or severe cytopenia
•3. Documented peripheral polyneuropathy. Intolerance to lactose
•4. Diarrhoea of unknown origin within 6 months prior to visit 1 or history of significant gastrointestinal disorders
•5. Cataracts or a known risk of cataract formation
•6. Severe renal insufficiency
•7. Active intercurrent diseases, such as human immunodeficiency virus (HIV) or hepatitis B or C
•8. Dependence or current abuse of drugs or alcohol
•9. Suspected hypersensitivity to miglustat or any of the excipients
•10. Patients who have previously received miglustat
•11. Pregnancy or breast-feeding
•12. Patients who refuse to use a reliable contraceptive method throughout the study and during the three months following the discontinuation of miglustat
•13. Patients receiving treatment with another investigational product or have received an investigational product in the 3 months prior to baseline

发起方

Aragon Institute of Health Sciences [Instituto Aragonés de Ciencias de la Salud] (Spain)