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 - PB-102-F01: PRX-102 for ERT in patients with Fabry disease

Protalix Ltd.0 个研究点目标入组 18 人开始时间: 2013年1月7日最近更新: 2015年8月4日

适应症

试验速览

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•1. Symptomatic adult Fabry patients (=18 yrs, males and females)
•2. 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)
•3. Females: historical genetic test results consistent with Fabry mutations
•4. Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
•5. 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
•6. Chronic kidney disease - stages 1 or 2 (CKD1 or 2) (Appendix 7) with proteinuria > 200 mg/g protein-to-creatinine ratio measured in a Spot urine sample or equivalent demonstrated in 2 separate samples (one sample in screening visit and the other from historical data)
•7. The patient signs informed consent
•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: 0
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 17
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•1. Participation in any trial of an investigational drug within 30 days prior to study screening
•2. Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
•3. History of dialysis or renal transplantation
•4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
•5. Severe myocardial fibrosis by MRI (=2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009)
•6. History of clinical stroke
•7. Pregnant or nursing
•8. Presence of HIV and/or HBsAg and/or Hepatitis C infections
•9. Known allergies to ERT
•10. Known allergy to Gadolinium based contrast agents
•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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