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

This study is currently recruiting participants.
Verified September 2017 by Protalix
Sponsor:
ClinicalTrials.gov Identifier:
NCT02795676
First Posted: June 10, 2016
Last Update Posted: October 2, 2017
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.
Information provided by (Responsible Party):
Protalix
  Purpose
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 Intervention Phase
Fabry Disease Biological: PRX-102 (pegunigalsidase alfa) Biological: agalsidase beta Phase 3

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Protalix:

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


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

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

  • 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 ]

Estimated Enrollment: 78
Study Start Date: June 2016
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: March 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

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.


Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT02795676


Contacts
Contact: Raul Chertkoff, MD +972-4-9028100 raul@protalix.com
Contact: Mali Szlaifer, MS malis@protalix.com

  Hide Study Locations
Locations
United States, Alabama
UAB Medicine Recruiting
Birmingham, Alabama, United States, 35233
Contact: Eric L Wallace, MD    205-975-9676    ericlwallace@uab.edu   
Principal Investigator: Eric L Wallace, MD         
United States, California
University of California Los Angeles Not yet recruiting
Los Angeles, California, United States, 90024
Contact: Anjay Rastogi    310-954-2692    drrastogioffice@mednet.ucla.edu   
Principal Investigator: Anjay Rastogi         
University of California Irvine Center Recruiting
Orange, California, United States, 92868
Contact: Virginia Kimonis    714-456-5791    vkimonis@uci.edu   
Principal Investigator: Virginia Kimonis         
University of California Davis Health System Not yet recruiting
Sacramento, California, United States, 95817
Contact: Madelena Martin    916-703-0203    bcgmartin@ucdavis.edu   
Principal Investigator: Madelena Martin         
University of California San Diego Recruiting
San Diego, California, United States, 92093
Contact: Bruce Barshop    619-543-5237    bbarshop@ucsd.edu   
Principal Investigator: Bruce Barshop         
United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: William Wilcox, MD    404-727-5624    william.wilcox@emory.edu   
Principal Investigator: William Wilcox, MD         
United States, Iowa
University of Iowa Hosptials and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Myrl Holida, PA    319-356-2007    myrl-holida@uiowa.edu   
Contact: Bernat John, MD    319-367-5630    john-bernat@uiowa.edu   
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    akaraa@mgh.harvard.edu   
Principal Investigator: Amel Karaa         
United States, Michigan
Infusion Associates Recruiting
Grand Rapids, Michigan, United States, 49525
Contact: Kahn Nedd, MD    616-954-0600    khannedd@yahoo.com   
Principal Investigator: Kahn Nedd, MD         
United States, Minnesota
Regents of the University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Chester Whitley    612-625-7422    whitley@umn.edu   
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 Not yet recruiting
Durham, North Carolina, United States, 27705
Contact: Marie McDonald    919-681-1982    marie.mcdonald@duke.edu   
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    rob.hopkin@cchmc.org   
Principal Investigator: Robert Hopkin, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Gerard Vockley, MD, PhD    412-692-7746    gerard.vockley@chp.edu   
Principal Investigator: Gerard Vockley, Md, PhD         
United States, Texas
Institute of Metabolic Disease, Baylor Healthcare Recruiting
Dallas, Texas, United States, 75226
Contact: Raphael Schiffmann, MD, MHSc    214-820-4533    raphael.schiffmann@baylorhealth.edu   
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    nicola.longo@hsc.utah.edu   
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    ogokeralpan@oandoalpan.com   
Contact: Thao-Chi Vo    571-308-1927    tvo@oandoalpan.com   
Principal Investigator: Ozlem Goker-Alpan, MD         
United States, Washington
University of Washington Not yet recruiting
Seattle, Washington, United States, 98105
Contact: Ron Scott    206-543-0937    crscott@u.washington.edu   
Principal Investigator: Ron Scott         
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226-3596
Contact: William Rhead    414-266-3345    wrhead@mcw.edu   
Principal Investigator: William Rhead         
Australia, Victoria
Royal Melbourne Hospital Recruiting
Parkville, Victoria, Australia, 3050
Contact: Kathleen Nicholls, MD    +61393427000    kathy.nicholls@mh.org.au   
Principal Investigator: Kathleen Nicholls, MD         
Belgium
UZ Antwerpen Recruiting
Edegem, Belgium, 2650
Contact: Francois Eyskens, MD    +32 38213810    francois.eyskens@uza.be   
Principal Investigator: Francois Eyskens, MD         
Canada, Nova Scotia
Capital District Health Authority Recruiting
Halifax, Nova Scotia, Canada, B3H 1V8
Contact: Michael L West, MD    +9024734023    mlwest@dal.ca   
Principal Investigator: Michael L West, Md         
Czechia
Vseobecna fakultni nemocnice v Praze Recruiting
Prague, Czech Republic, Czechia, 12808
Contact: Ales Linhart, MD    +420224962605    alinh@lf1.cuni.cz   
Principal Investigator: Ales Linhart, MD         
Germany
Universitaetsklinikum Wuerzburg Recruiting
Würzburg, Bayern, Germany, 97080
Contact: Christoph Wanner, MD    +49 931 20139030    wanner_c@klinik.uni-wuerzburg.de   
Principal Investigator: Christoph Wanner, MD         
Hungary
Semmelweis Egyetem Recruiting
Budapest, Hungary, 1083
Contact: Maria J Molnar, MD    +36206632513    molnarmj@gmail.com   
Principal Investigator: Maria J Molnar, MD         
Italy
Azienda Ospedaliero-Universitaria Careggi (SOD Nefrologia) Recruiting
Firenze, Italy, 50134
Contact: Eugenio E Minetti    0039 0557946997    minettie@aou-careggi.toscana.it   
Principal Investigator: Eugenio E Minetti         
Policlinico Universitario Agostino Gemelli, L.go A. Gemelli Recruiting
Roma, Italy, 00168
Contact: Raffaele Manna    0039 3483220416    raffaele.manna@policlinicogemelli.it   
Contact    0039 063503743      
Principal Investigator: Raffaele Manna         
Netherlands
Academisch Medisch Centrum Recruiting
Amsterdam, Netherlands, 1105 AZ
Contact: Mirjam Langeveld, MD, PhD    31205666071    m.langeveld@amc.nl   
Principal Investigator: Mirjam Langeveld, MD, PhD         
Norway
Haukeland University Hospital Klinisk Forskningspost Recruiting
Bergen, Norway, 5021
Contact: Camilla Tøndel, MD    +4755973859    camilla.tondel@helse-bergen.no   
Principal Investigator: Camilla Tøndel, MD         
Paraguay
Hematology and Clinical Research Private Institute Not yet recruiting
Asuncion, Paraguay, 1141
Contact: Derlis Emilio Gonzalez Rodriguez, MD    +59 521420888    gderlis@conexion.com.py   
Principal Investigator: Derlis Emilio Gonzales Rodriguez, MD         
Slovenia
General Hospital Slovenj Gradec Recruiting
Slovenj Gradec, Slovenia, 2380
Contact: Bojan Vujkovac    386 (0)28823708    bojan.vujkovac@guest.arnes.si   
Principal Investigator: Bojan Vujkovac         
Spain
Hospital de Dia Quiron Zaragoza Recruiting
Zaragoza, Spain, 50012
Contact: Beatriz Escuder    +34676605758    bea.escuderazuara@gmail.com   
Principal Investigator: Pilar Giraldo, MD         
Turkey
Department and Laboratory of Pediatric Metabolic Disorders Recruiting
Ankara, Turkey, 6500
Contact: Fatih Ezgu, MD    +90 5326853697    fezgu@gazi.edu.tr   
Principal Investigator: Fatih Ezgu, MD         
Ege Universty Medical Faculty Recruiting
Izmir, Turkey, 35100
Contact: Sema Kalkan Ucar, MD    +905057797312    semakalkan@hotmail.com   
Principal Investigator: Sema Kalkan Ucar, MD         
United Kingdom
Addenbrooke's Hospital Recruiting
Cambridge, United Kingdom, CB2 0QQ
Contact: Patrick Deegan, MD    +441223245151    patrick.deegan@addenbrookes.nhs.uk   
Principal Investigator: Patrick Deegan, MD         
University College London Hospitals Recruiting
London, United Kingdom, NW1 2PG
Contact: Robin Lachmann, MD    +442078298778    robin.lachmann@uclh.nhs.uk   
Principal Investigator: Robin Lachmann, MD         
The Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Derralynn Hughes, MD    4402077940500 ext 22496    rmgvdah@ucl.ac.uk   
Principal Investigator: Derralynn Hughes, MD         
Salford Royal NHS Foundation Trust Recruiting
Salford, United Kingdom, M6 8HD
Contact: Ana Jovanovic, MD    +441612064365    ana.jovanovic@srft.nhs.uk   
Principal Investigator: Ana Jovanovic, MD         
Sponsors and Collaborators
Protalix
Investigators
Study Director: Raul Chertkoff, MD Protalix Ltd
  More Information

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT02795676     History of Changes
Other Study ID Numbers: PB-102-F20
First Submitted: June 2, 2016
First Posted: June 10, 2016
Last Update Posted: October 2, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Protalix:
Glomerular filtration rate
Proteinuria
PRX-102
pegunigalsidase alfa
Fabry Disease

Additional relevant MeSH terms:
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