A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta - BALANCE study

Protalix Ltd0 个研究点目标入组 5 人开始时间: 待定最近更新: 2024年2月28日

研究概览

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•1. Symptomatic adult Fabry disease patients, age 18-60 years
•a. Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels
•b. One or more of the described characteristic features of Fabry disease:
•i. neuropathic pain,
•ii. cornea verticillata,
•iii. clustered angiokeratoma
•2. Screening eGFR by CKD-EPI equation 40 to 90 mL/min/1.73 m2
•3. Linear negative slope of eGFR of * 2 mL/min/1.73 m2 based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit)
•4. Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months
•5. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.

•1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
•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. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
•7. Congestive heart failure NYHA Class IV
•8. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
•9. Known history of hypersensitivity to Gadolinium contrast agent
•10. Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
•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

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