MedDRA version: 20.0 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. Completion of study PB-102-F20, PB-102-F30 or completed at least 48 months in study PB-102-F03 2. The patient signs informed consent 3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a highly effective medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include combined (estrogen- and progestogen-containing) hormonal products associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence which is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject. Contraception should be used for 1 month after treatment termination. 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 110 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient compliance with the requirements of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the ongoing safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa (PRX-102) every other week in adult Fabry patients who have successfully completed studies PB-102-F20, PB-102-F30 or completed at least 48 months in study PB-102-F03;Secondary Objective: N/A;Primary end point(s): SAFETY ENDPOINTS: Changes from baseline in: *Clinical laboratory tests *Physical examination *Assessment of the injection site *Electrocardiography (ECG) *Treatment-emergent adverse events (TEAE) *Ability to taper off infusion pre-medication at the start of the study *Requirement for use of pre-medication overall to manage infusion reactions *Treatment-emergent anti-pegunigalsidase alfa antibodies;Timepoint(s) of evaluation of this end point: Patients who received at least one dose of study drug will be included in the safety analysis. Results of safety evaluations (including TEAEs, injection site reactions, physical examinations, ECG, and laboratory analyses, anti-drug antibodies) will be summarized and described. No formal statistical analyses will be performed. The study endpoints will be evaluated by various summary analyses by study visit and by change from baseline data for each efficacy endpoint. Safety and efficacy endpoints will be compared to baseline using summary statistics. Data will not be analysed by inferential statistics. An interim analysis may be performed for administrative purposes during the course of the study; available efficacy and safety parameters will be summarized using descriptive statistics. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): EFFICACY ENDPOINTS: *Estimated glomerular filtration rate (eGFRCKD-EPI) *Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) *Plasma Lyso-Gb3 concentration *Plasma Gb3 concentration *Protein/Creatinine ratio, spot urine test (UPCR) *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) *Fabry disease clinical events ;Timepoint(s) of evaluation of this end point: N/A | — |
Countries
Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, Denmark, Germany, Hungary, Italy, Netherlands, Norway, Slovenia, Spain, Switzerland, Turkey, United Kingdom, United States
Contacts
Public ContactRaul Chertkoff
Protalix Ltd.
Outcome results
None listed