A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da USP0 个研究点开始时间: 2015年12月21日最近更新: 2023年5月29日

适应症

试验速览

暂无简介。

•Symptomatic adult Fabry patients (?18 yrs; Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein); Females: historical genetic test results consistent with Fabry mutations; Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit; Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti PRX-102 antibody test; Chronic kidney disease - stages 1 or 2 (CKD1 or 2) (Appendix 7) with proteinuria > 200 mg/g protein-to-creatinine ratio or equivalent, measured in a Spot urine sample, demonstrated in at least one of 2 separate samples (1 sample at screening visit and the other from historical data); The patient signs informed consent; Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method

•Participation in any trial of an investigational drug within 30 days prior to study screening; Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7); History of dialysis or renal transplantation; Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening ; Severe myocardial fibrosis by MRI (?2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009); History of clinical stroke; Pregnant or nursing; Presence of HIV and/or HBsAg and/or Hepatitis C infections; Known allergies to ERT; Known allergy to Gadolinium based contrast agents;Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient’s compliance with the requirements of the study

发起方

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da USP