Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion to Adult Fabry Patients

• Conditions: Fabry disease; MedDRA version: 16.1Level: 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-ES
• Lead Sponsor: Protalix Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-09-05
• Last Update Posted: 4 August 2015

Recruitment & Eligibility

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

Inclusion Criteria

1.Symptomatic adult Fabry patients (> or = to 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 at least one of 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:
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 (> or = to 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 patients compliance with the requirements of the study

Study & Design

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