A clinical study in patients with Fabry disease to assess the safety, tolerability, and the body processing of the medication PRX-102, which will be given as an infusio

• Conditions: Fabry disease; MedDRA version: 17.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2012-004786-40-GB
• Lead Sponsor: Protalix Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-01-07
• Last Update Posted: 4 August 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified