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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02795676
Recruitment Status : Active, not recruiting
First Posted : June 10, 2016
Last Update Posted : October 30, 2019
Sponsor:
Information provided by (Responsible Party):
Protalix

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 : October 2021
Estimated Study Completion Date : May 2022

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
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

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


Locations
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United States, Alabama
UAB Medicine
Birmingham, Alabama, United States, 35233
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, Arkansas
Arkansas Children's hospital
Little Rock, Arkansas, United States, 30020-8341
United States, California
University of California Los Angeles
Los Angeles, California, United States, 90024
University of California Irvine Center
Orange, California, United States, 92868
University of California San Diego
San Diego, California, United States, 92093
United States, Florida
University of Florida, Department of Pediatrics/Genetics & Metabolism
Gainesville, Florida, United States, 32608
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Iowa
University of Iowa Hosptials and Clinics
Iowa City, Iowa, United States, 52242
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Michigan
Infusion Associates
Grand Rapids, Michigan, United States, 49525
United States, Minnesota
Regents of the University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, New York
New York University Medical Center
New York, New York, United States, 10016
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Tennessee
The University of Tennessee
Memphis, Tennessee, United States, 38163
United States, Texas
Institute of Metabolic Disease, Baylor Healthcare
Dallas, Texas, United States, 75226
United States, Utah
Eccles Primary Children's Outpatient Services Building
Salt Lake City, Utah, United States, 84132
United States, Virginia
O+O Alpan LLC
Fairfax, Virginia, United States, 22030
United States, West Virginia
West Virginia University
Morgantown, West Virginia, United States, 26506
United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226-3596
Argentina
Instituto de Nefrologia Pergamino SRL
Pergamino, Buenos Aires, Argentina, 2700
Australia, Victoria
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Belgium
UZ Antwerpen
Edegem, Belgium, 2650
Brazil
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
Canada, Nova Scotia
Capital District Health Authority
Halifax, Nova Scotia, Canada, B3H 1V8
Czechia
Vseobecna fakultni nemocnice v Praze
Prague, Czech Republic, Czechia, 12808
Finland
Turku University Central Hospital
Turku, Finland, 20520
France
Hôpital Raymond Poincaré
Paris, France, 92380
Germany
Universitaetsklinikum Wuerzburg
Würzburg, Bayern, Germany, 97080
Hungary
Semmelweis Egyetem
Budapest, Hungary, 1083
Italy
Azienda Ospedaliero-Universitaria Careggi (SOD Nefrologia)
Firenze, Italy, 50134
Azienda Ospedaliera Universitaria "Federico II"
Napoli, Italy
Policlinico Universitario Agostino Gemelli, L.go A. Gemelli
Roma, Italy, 00168
Netherlands
Academisch Medisch Centrum
Amsterdam, Netherlands, 1105 AZ
Norway
Haukeland University Hospital Klinisk Forskningspost
Bergen, Norway, 5021
Paraguay
Hematology and Clinical Research Private Institute
Asuncion, Paraguay, 1141
Slovenia
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia, 2380
Spain
Hospital de Dia Quiron Zaragoza
Zaragoza, Spain, 50012
Switzerland
Klinik und Poliklinik für Innere Medizin UniversitätsSpital Zürich
Zürich, Switzerland
Turkey
Department and Laboratory of Pediatric Metabolic Disorders
Ankara, Turkey, 6500
Ege Universty Medical Faculty
Izmir, Turkey, 35100
United Kingdom
Institute of Metabolism and Systems Research
Edgbaston, Birmingham, United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom, CB2 0QQ
University Hospital of Wales
Cardiff, United Kingdom, CF14 4XW
University College London Hospitals
London, United Kingdom, NW1 2PG
The Royal Free Hospital
London, United Kingdom, NW3 2QG
Salford Royal NHS Foundation Trust
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
Protalix
Investigators
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Study Director: Raul Chertkoff, MD Protalix Ltd.
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Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT02795676    
Other Study ID Numbers: PB-102-F20
First Posted: June 10, 2016    Key Record Dates
Last Update Posted: October 30, 2019
Last Verified: October 2019
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:
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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
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors