Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
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ClinicalTrials.gov Identifier: NCT03018730 |
Recruitment Status :
Completed
First Posted : January 12, 2017
Last Update Posted : January 5, 2021
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Condition or disease | Intervention/treatment | Phase |
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Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa) | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 22 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) |
Actual Study Start Date : | February 23, 2017 |
Actual Primary Completion Date : | December 17, 2019 |
Actual Study Completion Date : | December 17, 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: PRX-102
PRX-102 infusion every 2 weeks
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Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
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- Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Throughout the 12 months study ]
- Mean annualised change in eGFR [ Time Frame: Every 4 weeks for 12 months ]Mean annualised change in eGFR
- Left Ventricular Mass Index (g/m2) preferably by MRI [ Time Frame: Every 6 months for 12 months ]Left Ventricular Mass Index (g/m2) preferably by MRI (ECG can be used as an alternative)
- Plasma Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
- Plasma Gb3 [ Time Frame: Every 3 months for 12 months ]
- Urine Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
- Protein/Creatinine ratio [ Time Frame: Every 3 months for 12 months ]Protein/Creatinine ratio spot urine test
- Frequency of pain medication use [ Time Frame: Every 2 weeks for 12 months ]
- Exercise tolerance (Stress Test) [ Time Frame: Every 6 months for 12 months ]
- Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 3 months for 12 months ]
- Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 12 months ]
- Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 12 months ]

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Ages Eligible for Study: | 18 Years to 60 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age: 18-60 years
- A documented diagnosis of Fabry disease
- 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
- 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
- Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months
- eGFR ≥ 40 ml/min/1.73 m2 by CKD-EPI equation
- Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years
- 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:
- History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa
- History of renal dialysis or transplantation
- 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)
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
- Known history of hypersensitivity to Gadolinium contrast agent
- Females who are pregnant, planning to become pregnant during the study, or are breast feeding
- Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening
- Congestive heart failure NYHA Class IV
- Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03018730
Australia, Victoria | |
Royal Melbourne Hospital | |
Parkville, Victoria, Australia, 3050 | |
Canada, Nova Scotia | |
Capital Health | |
Halifax, Nova Scotia, Canada, B3H 1V8 | |
Czechia | |
Vseobecna fakultni nemocnice v Praze | |
Prague, Czechia | |
Germany | |
Universitaetsklinikum Wuerzburg | |
Wurzburg, Germany | |
Netherlands | |
Academisch Medisch Centrum | |
Amsterdam, Netherlands | |
Norway | |
Helse Bergen HF Haukeland Universitetssykehus | |
Bergen, Norway | |
Slovenia | |
General Hospital Slovenj Gradec | |
Slovenj Gradec, Slovenia, SI-2380 | |
Spain | |
Hospital de Dia Quiron Zaragoza | |
Zaragoza, Spain, 50012 | |
United Kingdom | |
Institute of Metabolism and Systems Research | |
Edgbaston, Birmingham, United Kingdom, B15 2UR | |
Addenbrooke's Hospital | |
Cambridge, United Kingdom, CB2 0QQ | |
The Royal Free Hospital | |
London, United Kingdom, NW3 2QG | |
Salford Royal NHS Foundation Trust | |
Salford, United Kingdom, M6 8HD |
Study Director: | Raul Chertkoff, MD | Protalix Ltd. |
Responsible Party: | Protalix |
ClinicalTrials.gov Identifier: | NCT03018730 |
Other Study ID Numbers: |
PB-102-F30 |
First Posted: | January 12, 2017 Key Record Dates |
Last Update Posted: | January 5, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Fabry disease Enzyme-Replacement Therapy pegunigalsidase alfa PRX-102 alpha galactosidase-A |
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders |
Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |