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Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function (BALANCE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02795676
Recruitment Status : Recruiting
First Posted : June 10, 2016
Last Update Posted : March 28, 2019
Information provided by (Responsible Party):

Brief Summary:
This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with 1mg/kg agalsidase beta or to treatment with 1 mg/kg of PRX-102. The identity of the enzyme will be blinded to the patient and the investigator. Patients will receive intravenous infusions every two weeks. Patients will be randomized in a 2:1 ratio of PRX-102 to agalsidase beta. Randomization will be stratified by urinary protein to creatinine ratio (UPCR) of < or ≥ 1 g/g by spot urine sample. No more than 50% of the patients will be female.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: PRX-102 (pegunigalsidase alfa) Biological: agalsidase beta Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 78 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta
Study Start Date : June 2016
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : June 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Tests

Arm Intervention/treatment
Experimental: PRX-102 (pegunigalsidase alfa)
PRX-102 infusion every 2 weeks
Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
  • pegunigalsidase alfa
  • Recombinant human alpha galactosidase-A

Active Comparator: agalsidase beta
agalsidase beta infusion every 2 weeks
Biological: agalsidase beta
agalsidase beta 1 mg/kg every 2 weeks
Other Name: Fabrazyme

Primary Outcome Measures :
  1. eGFR Slope [ Time Frame: Every month for 2 years ]
    eGFR Slope

Secondary Outcome Measures :
  1. Left Ventricular Mass Index (g/m2) by MRI [ Time Frame: Every 12 months for 2 years ]
  2. Plasma Lyso-Gb3 [ Time Frame: Every 3 months for 2 years ]
  3. Plasma Gb3 [ Time Frame: Every 3 months for 2 years ]
  4. Urine Lyso-GB3 [ Time Frame: Every 6 weeks for 2 years ]
  5. Protein/creatinine ratio [ Time Frame: Every 4 months for 24 months ]
  6. Frequency of pain medication use [ Time Frame: Every 2 weeks for 2 years ]
  7. Exercise tolerance (Stress Test) [ Time Frame: Every year for 2 years ]
  8. Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 6 months for 2 years ]
  9. Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 2 years ]
  10. Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 2 years ]

Other Outcome Measures:
  1. PRX-102 pharmacokinetics [ Time Frame: Every 6 months for 2 years ]
    PRX-102 pharmacokinetics parameters

  2. Anti-drug IgG antibodies [ Time Frame: Every 2 weeks for 1 month, then every month for the first 6 months and every 3 month until the end of the study ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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:

  • Symptomatic adult Fabry disease patients, age 18-60 years

    1. Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels and one or more of the characteristic features of Fabry disease

      i. neuropathic pain

      ii. cornea verticillata

      iii. clustered angiokeratoma

    2. Females:

      a. historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:

      i. neuropathic pain

      ii. cornea verticillata

      iii. clustered angiokeratoma

      b. or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease

      i. neuropathic pain

      ii. cornea verticillata

      iii. clustered angiokeratoma

  • Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
  • Linear negative slope of eGFR based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit) of ≥ 2 mL/min/1.73 m²/year
  • Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months.
  • Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.

Exclusion Criteria:

  • History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
  • Known non-pathogenic Fabry mutations
  • 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
  • Patient with a screening eGFR value between 91-120 mL/min/1.73 m², having an historical eGFR value higher than 120 mL/min/1.73 m² (during 9 to 18 months before screening)
  • Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
  • Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
  • Congestive heart failure NYHA Class IV
  • Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
  • Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of pre-medication
  • Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
  • 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

Information from the National Library of Medicine

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 identifier (NCT number): NCT02795676

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Contact: Raul Chertkoff, MD +972-4-9028100
Contact: Mali Szlaifer, MS

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United States, Alabama
UAB Medicine Recruiting
Birmingham, Alabama, United States, 35233
Contact: Eric L Wallace, MD    205-975-9676   
Principal Investigator: Eric L Wallace, MD         
United States, Arizona
Phoenix Children's Hospital Not yet recruiting
Phoenix, Arizona, United States, 85016
Contact: Kristin E. Lindstrom, MD    602-933-4363   
Principal Investigator: Kristin E. Lindstrom, MD         
United States, Arkansas
Arkansas Children's hospital Recruiting
Little Rock, Arkansas, United States, 30020-8341
Contact: Thomas Burrow, MD    501-364-2966   
Principal Investigator: Thomas Burrow, MD         
United States, California
University of California Los Angeles Recruiting
Los Angeles, California, United States, 90024
Contact: Anjay Rastogi, MD    310-954-2692   
Principal Investigator: Anjay Rastogi, MD         
University of California Irvine Center Recruiting
Orange, California, United States, 92868
Contact: Virginia Kimonis    714-456-5791   
Principal Investigator: Virginia Kimonis         
University of California San Diego Recruiting
San Diego, California, United States, 92093
Contact: Bruce Barshop    619-543-5237   
Principal Investigator: Bruce Barshop         
United States, Florida
University of Florida, Department of Pediatrics/Genetics & Metabolism Not yet recruiting
Gainesville, Florida, United States, 32608
Contact: Maegawa Gustavo, MD, PhD    352-294-5559   
Principal Investigator: Maegawa Gustavo, MD, PhD         
United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: William Wilcox, MD    404-727-5624   
Principal Investigator: William Wilcox, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Joel Charrow, MD    773-880-4462   
Principal Investigator: Joel Charrow, MD         
United States, Iowa
University of Iowa Hosptials and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Myrl Holida, PA    319-356-2007   
Contact: Bernat John, MD    319-367-5630   
Principal Investigator: Myrl Holida, PA         
Principal Investigator: Bernat John, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Amel Karaa    617-726-1561   
Principal Investigator: Amel Karaa         
United States, Michigan
Infusion Associates Recruiting
Grand Rapids, Michigan, United States, 49525
Contact: Kahn Nedd, MD    616-954-0600   
Principal Investigator: Kahn Nedd, MD         
United States, Minnesota
Regents of the University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Chester Whitley    612-625-7422   
Principal Investigator: Chester Whitley         
United States, New York
New York University Medical Center Recruiting
New York, New York, United States, 10016
Contact: Heather Lau, MD    212-263-8344      
Principal Investigator: Heather Lau, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27705
Contact: Marie McDonald    919-681-1982   
Principal Investigator: Marie McDonald         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Robert Hopkin, MD    513-636-4760   
Principal Investigator: Robert Hopkin, MD         
United States, Oregon
Oregon Health & Science University Not yet recruiting
Portland, Oregon, United States, 97239
Contact: Amy C. Yang, MD    503-418-2385   
Principal Investigator: Amy C. Yang, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Gerard Vockley, MD, PhD    412-692-7746   
Principal Investigator: Gerard Vockley, Md, PhD         
United States, Tennessee
The University of Tennessee Not yet recruiting
Memphis, Tennessee, United States, 38163
Contact: John L. Jefferies, MD    901-448-5750   
Principal Investigator: John L. Jefferies, MD         
United States, Texas
Institute of Metabolic Disease, Baylor Healthcare Recruiting
Dallas, Texas, United States, 75226
Contact: Raphael Schiffmann, MD, MHSc    214-820-4533   
Principal Investigator: Raphael Schiffmann, MD, MHSc         
United States, Utah
Eccles Primary Children's Outpatient Services Building Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Nicola Longo, MD, PhD    801-587-3605   
Principal Investigator: Nicola Longo, MD, PhD         
United States, Virginia
O+O Alpan LLC Recruiting
Fairfax, Virginia, United States, 22030
Contact: Ozlem Goker-Alpan, MD    571-308-1900   
Contact: Thao-Chi Vo    571-308-1927   
Principal Investigator: Ozlem Goker-Alpan, MD         
United States, West Virginia
West Virginia University Recruiting
Morgantown, West Virginia, United States, 26506
Contact: Tarachandra Narumanchi, MD    304-293-7332   
Principal Investigator: Tarachandra Narumanchi, MD         
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226-3596
Contact: William Rhead    414-266-3345   
Principal Investigator: William Rhead         
Instituto de Nefrologia Pergamino SRL Recruiting
Pergamino, Buenos Aires, Argentina, 2700
Contact: Norberto R Antongiovanni, MD    +54 2477433928   
Principal Investigator: Norberto R Antongiovanni, MD         
Australia, Victoria
Royal Melbourne Hospital Recruiting
Parkville, Victoria, Australia, 3050
Contact: Kathleen Nicholls, MD    +61393427000   
Principal Investigator: Kathleen Nicholls, MD         
UZ Antwerpen Recruiting
Edegem, Belgium, 2650
Contact: Francois Eyskens, MD    +32 38213810   
Principal Investigator: Francois Eyskens, MD         
Hospital de Clínicas de Porto Alegre Recruiting
Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
Contact: Robert Giugliani, MD    +55 5133515369   
Principal Investigator: Robert Giugliani, MD         
Canada, Nova Scotia
Capital District Health Authority Recruiting
Halifax, Nova Scotia, Canada, B3H 1V8
Contact: Michael L West, MD    +9024734023   
Principal Investigator: Michael L West, Md         
Vseobecna fakultni nemocnice v Praze Recruiting
Prague, Czech Republic, Czechia, 12808
Contact: Ales Linhart, MD    +420224962605   
Principal Investigator: Ales Linhart, MD         
Universitaetsklinikum Wuerzburg Not yet recruiting
Würzburg, Bayern, Germany, 97080
Contact: Christoph Wanner, MD    +49 931 20139030   
Principal Investigator: Christoph Wanner, MD         
Semmelweis Egyetem Recruiting
Budapest, Hungary, 1083
Contact: Maria J Molnar, MD    +36206632513   
Principal Investigator: Maria J Molnar, MD         
Azienda Ospedaliero-Universitaria Careggi (SOD Nefrologia) Recruiting
Firenze, Italy, 50134
Contact: Eugenio E Minetti    0039 0557946997   
Principal Investigator: Eugenio E Minetti         
Azienda Ospedaliera Universitaria "Federico II" Recruiting
Napoli, Italy
Contact: Pisani Antonio, MD    0039 0817464521   
Principal Investigator: Pisani Antonio, MD         
Policlinico Universitario Agostino Gemelli, L.go A. Gemelli Recruiting
Roma, Italy, 00168
Contact: Raffaele Manna    0039 3483220416   
Contact    0039 063503743      
Principal Investigator: Raffaele Manna         
Academisch Medisch Centrum Recruiting
Amsterdam, Netherlands, 1105 AZ
Contact: Mirjam Langeveld, MD, PhD    31205666071   
Principal Investigator: Mirjam Langeveld, MD, PhD         
Haukeland University Hospital Klinisk Forskningspost Recruiting
Bergen, Norway, 5021
Contact: Camilla Tøndel, MD    +4755973859   
Principal Investigator: Camilla Tøndel, MD         
Hematology and Clinical Research Private Institute Not yet recruiting
Asuncion, Paraguay, 1141
Contact: Derlis Emilio Gonzalez Rodriguez, MD    +59 521420888   
Principal Investigator: Derlis Emilio Gonzales Rodriguez, MD         
General Hospital Slovenj Gradec Recruiting
Slovenj Gradec, Slovenia, 2380
Contact: Bojan Vujkovac    386 (0)28823708   
Principal Investigator: Bojan Vujkovac         
Hospital de Dia Quiron Zaragoza Recruiting
Zaragoza, Spain, 50012
Contact: Beatriz Escuder    +34676605758   
Principal Investigator: Pilar Giraldo, MD         
Klinik und Poliklinik für Innere Medizin UniversitätsSpital Zürich Recruiting
Zürich, Switzerland
Contact: Albina N Oberärztin, MD    +41 (0)44 255 10 54   
Principal Investigator: Albina N Oberärztin, MD         
Department and Laboratory of Pediatric Metabolic Disorders Recruiting
Ankara, Turkey, 6500
Contact: Fatih Ezgu, MD    +90 5326853697   
Principal Investigator: Fatih Ezgu, MD         
Ege Universty Medical Faculty Recruiting
Izmir, Turkey, 35100
Contact: Sema Kalkan Ucar, MD    +905057797312   
Principal Investigator: Sema Kalkan Ucar, MD         
United Kingdom
Institute of Metabolism and Systems Research Recruiting
Edgbaston, Birmingham, United Kingdom
Contact: Tarekegn G Hiwot, MD    +44 (0)121 371 6982   
Contact: Tarekegn G Hiwot, MD    +44 (0)121 371 6982   
Principal Investigator: Tarekegn G Hiwot, MD         
Addenbrooke's Hospital Recruiting
Cambridge, United Kingdom, CB2 0QQ
Contact: Patrick Deegan, MD    +441223245151   
Principal Investigator: Patrick Deegan, MD         
University Hospital of Wales Recruiting
Cardiff, United Kingdom, CF14 4XW
Contact: Cole Duncan, MD    +44(0)29 20748359   
Principal Investigator: Cole Duncan, MD         
University College London Hospitals Recruiting
London, United Kingdom, NW1 2PG
Contact: Robin Lachmann, MD    +442078298778   
Principal Investigator: Robin Lachmann, MD         
The Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Derralynn Hughes, MD    4402077940500 ext 22496   
Principal Investigator: Derralynn Hughes, MD         
Salford Royal NHS Foundation Trust Recruiting
Salford, United Kingdom, M6 8HD
Contact: Ana Jovanovic, MD    +441612064365   
Principal Investigator: Ana Jovanovic, MD         
Sponsors and Collaborators
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Study Director: Raul Chertkoff, MD Protalix Ltd

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Responsible Party: Protalix Identifier: NCT02795676     History of Changes
Other Study ID Numbers: PB-102-F20
First Posted: June 10, 2016    Key Record Dates
Last Update Posted: March 28, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Protalix:
Glomerular filtration rate
pegunigalsidase alfa
Fabry Disease

Additional relevant MeSH terms:
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Fabry Disease
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
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors