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

Not Applicable

• Conditions: -E752 Other sphingolipidosis; Other sphingolipidosis; E752

• Registration Number: PER-036-14
• Lead Sponsor: Protalix LTD,

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-10-10
• Last Update Posted: 4 September 2023

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 0

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

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
• Study Design: Not specified