None listed
Conditions
Interventions
PRX-102 0.2, 1 or 2 mg/kg, intravenously, every 2 weeks.
Duration of treatment: 12 weeks (7 infusions), with the option to be enrolled in an open-label extension study upon completion of the study.
Sponsors
Protalix LTD,
Eligibility
Sex/Gender
All
Age
18 Years to 90 Years
Inclusion criteria
Inclusion criteria: 1. Symptomatic adult Fabry patients (≥18 yrs) 2. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2nmol/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 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) 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.
Exclusion criteria
Exclusion criteria: 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
Countries
Australia, Israel, Paraguar, Peru, Serbia, Spain, United Kindgdom, United States
Contacts
Public ContactMarcela Toledo
SYNEOS HEALTH PERU S.R.L.
Outcome results
None listed