Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Protalix Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age: 18-60 years 2. A documented diagnosis of Fabry disease. 3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal according to laboratory range and one or more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata iii. Clustered angiokeratoma 4. Females: historical genetic test results consistent with Fabry mutations, or in the case of novel mutations a first degree male relative with Fabry disease, and one or more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata iii. Clustered angiokeratoma 5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months 6. eGFR = 40 ml/min/1.73 m2 by CKD-EPI equation 7. Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years 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 22 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa 2. History of renal dialysis or transplantation 3. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy) 4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening 5. Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB 6. Known history of hypersensitivity to Gadolinium contrast agent 7. Females who are pregnant, planning to become pregnant during the study, or are breast feeding 8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening 9. Congestive heart failure NYHA Class IV 10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa;Secondary Objective: N/A;Primary end point(s): SAFETY ENDPOINTS: Change from baseline in: • Clinical laboratory tests • Physical examination • Assessment of the injection site • Electrocardiogram • Treatment-emergent adverse events • Ability to taper off infusion premedication throughout the first 2 months of the study • Requirement for use of premedication overall to manage infusion reactions • Treatment-emergent anti-PRX-102 antibodies EFFICACY ENDPOINTS: • Mean annualised change in estimated glomerular filtration rate (eGFRCKD-EPI) • Left Ventricular Mass Index (g/m2) preferably by MRI (echocardiogram can be used as an alternative) • Plasma Lyso-Gb3 • Plasma Gb3 • Urine Lyso-Gb3 • Protein/Creatinine ratio spot urine test • Frequency of pain medication use • Exercise tolerance (Stress Test) • Short Form Brief Pain Inventory (BPI) • Mainz Severity Score Index (MSSI) • Quality of life EQ-5D-5L;Timepoint(s) of evaluation of this end point: As per protocol | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): N/A ;Timepoint(s) of evaluation of this end point: N/A | — |
Countries
Australia, Canada, Czech Republic, Germany, Netherlands, Norway, Slovenia, Spain, United Kingdom
Contacts
Public ContactRaul Chertkoff
Protalix Ltd.
Outcome results
None listed