An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)

Protalix Ltd.0 个研究点目标入组 22 人开始时间: 2017年10月17日最近更新: 2020年11月9日

研究概览

暂无简介。

•1. Age: 18-60 years
•2. A documented diagnosis of Fabry disease.
•3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal according to laboratory range and one or more of the characteristic features of Fabry disease
•i. Neuropathic pain
•ii. Cornea verticillata
•iii. Clustered angiokeratoma
•4. Females: historical genetic test results consistent with Fabry mutations, or in the case of novel mutations a first degree male relative with Fabry disease, and one or more of the characteristic features of Fabry disease
•i. Neuropathic pain
•ii. Cornea verticillata
•iii. Clustered angiokeratoma
•5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months
•6. eGFR = 40 ml/min/1.73 m2 by CKD-EPI equation
•7. Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years
•8. 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
•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 22
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa
•2. History of renal dialysis or transplantation
•3. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
•4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
•5. Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
•6. Known history of hypersensitivity to Gadolinium contrast agent that is
•not managed by the use of premedication
•7. Females who are pregnant, planning to become pregnant during the
•study, or are breast feeding
•8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening
•9. Congestive heart failure NYHA Class IV
•10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening
•11. 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