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

Description

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.

Study Start Date

June, 01 2016

Estimated Completion Date

June 2019

Interventions

  • Biological: agalsidase beta
  • Biological: PRX-102 (pegunigalsidase alfa)

Study ID

Protalix -- PB-102-F20

Status

Recruiting

Trial ID

NCT02795676

Study Type

Interventional

Trial Phase

Phase 3

Enrollment Quota

78

Sponsor

Protalix

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

Gender

All

Ages

18 Years to 60 Years

Accepts Healthy Volunteers

No

Study Locations and Contact Information (95)

Study Location Distance Name Phone Email
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles Amel Karaa 617-726-1561 akaraa@mgh.harvard.edu
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles Amel Karaa 617-726-1561 akaraa@mgh.harvard.edu
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles Amel Karaa 617-726-1561 akaraa@mgh.harvard.edu
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles Amel Karaa 617-726-1561 akaraa@mgh.harvard.edu
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles Amel Karaa 617-726-1561 akaraa@mgh.harvard.edu
New York University Medical Center - New York, New York 186.5 miles Heather Lau MD 212-263-8344 None
New York University Medical Center - New York, New York 186.5 miles Heather Lau MD 212-263-8344 None
New York University Medical Center - New York, New York 186.5 miles Heather Lau MD 212-263-8344 None
New York University Medical Center - New York, New York 186.5 miles Heather Lau MD 212-263-8344 None
New York University Medical Center - New York, New York 186.5 miles Heather Lau MD 212-263-8344 None
New York University Medical Center - New York, New York 186.5 miles Heather Lau MD 212-263-8344 None
OO Alpan LLC - Fairfax, Virginia 408.8 miles Ozlem GokerAlpan MD 571-308-1900 ogokeralpan@oandoalpan.com
OO Alpan LLC - Fairfax, Virginia 408.8 miles Ozlem GokerAlpan MD 571-308-1900 ogokeralpan@oandoalpan.com
OO Alpan LLC - Fairfax, Virginia 408.8 miles Ozlem GokerAlpan MD 571-308-1900 ogokeralpan@oandoalpan.com
OO Alpan LLC - Fairfax, Virginia 408.8 miles Ozlem GokerAlpan MD 571-308-1900 ogokeralpan@oandoalpan.com
OO Alpan LLC - Fairfax, Virginia 408.8 miles Ozlem GokerAlpan MD 571-308-1900 ogokeralpan@oandoalpan.com
OO Alpan LLC - Fairfax, Virginia 408.8 miles Ozlem GokerAlpan MD 571-308-1900 ogokeralpan@oandoalpan.com
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles Gerard Vockley MD PhD 412-692-7746 gerard.vockley@chp.edu
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles Gerard Vockley MD PhD 412-692-7746 gerard.vockley@chp.edu
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles Gerard Vockley MD PhD 412-692-7746 gerard.vockley@chp.edu
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles Gerard Vockley MD PhD 412-692-7746 gerard.vockley@chp.edu
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles Gerard Vockley MD PhD 412-692-7746 gerard.vockley@chp.edu
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles Gerard Vockley MD PhD 412-692-7746 gerard.vockley@chp.edu
Duke University Medical Center - Durham, North Carolina 608.4 miles Marie McDonald 919-681-1982 marie.mcdonald@duke.edu
Duke University Medical Center - Durham, North Carolina 608.4 miles Marie McDonald 919-681-1982 marie.mcdonald@duke.edu
Duke University Medical Center - Durham, North Carolina 608.4 miles Marie McDonald 919-681-1982 marie.mcdonald@duke.edu
Duke University Medical Center - Durham, North Carolina 608.4 miles Marie McDonald 919-681-1982 marie.mcdonald@duke.edu
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Robert Hopkin MD 513-636-4760 rob.hopkin@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Robert Hopkin MD 513-636-4760 rob.hopkin@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Robert Hopkin MD 513-636-4760 rob.hopkin@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Robert Hopkin MD 513-636-4760 rob.hopkin@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Robert Hopkin MD 513-636-4760 rob.hopkin@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Robert Hopkin MD 513-636-4760 rob.hopkin@cchmc.org
Infusion Associates - Grand Rapids, Michigan 738.6 miles Kahn Nedd MD 616-954-0600 khannedd@yahoo.com
Infusion Associates - Grand Rapids, Michigan 738.6 miles Kahn Nedd MD 616-954-0600 khannedd@yahoo.com
Infusion Associates - Grand Rapids, Michigan 738.6 miles Kahn Nedd MD 616-954-0600 khannedd@yahoo.com
Infusion Associates - Grand Rapids, Michigan 738.6 miles Kahn Nedd MD 616-954-0600 khannedd@yahoo.com
Infusion Associates - Grand Rapids, Michigan 738.6 miles Kahn Nedd MD 616-954-0600 khannedd@yahoo.com
Infusion Associates - Grand Rapids, Michigan 738.6 miles Kahn Nedd MD 616-954-0600 khannedd@yahoo.com
Emory University School of Medicine - Atlanta, Georgia 932.0 miles William Wilcox MD 404-727-5624 william.wilcox@emory.edu
Emory University School of Medicine - Atlanta, Georgia 932.0 miles William Wilcox MD 404-727-5624 william.wilcox@emory.edu
Emory University School of Medicine - Atlanta, Georgia 932.0 miles William Wilcox MD 404-727-5624 william.wilcox@emory.edu
Emory University School of Medicine - Atlanta, Georgia 932.0 miles William Wilcox MD 404-727-5624 william.wilcox@emory.edu
Emory University School of Medicine - Atlanta, Georgia 932.0 miles William Wilcox MD 404-727-5624 william.wilcox@emory.edu
Emory University School of Medicine - Atlanta, Georgia 932.0 miles William Wilcox MD 404-727-5624 william.wilcox@emory.edu
University of Iowa Hosptials and Clinics - Iowa City, Iowa 1,050.4 miles Myrl Holida PA 319-356-2007 myrl-holida@uiowa.edu
University of Iowa Hosptials and Clinics - Iowa City, Iowa 1,050.4 miles Myrl Holida PA 319-356-2007 myrl-holida@uiowa.edu
University of Iowa Hosptials and Clinics - Iowa City, Iowa 1,050.4 miles Myrl Holida PA 319-356-2007 myrl-holida@uiowa.edu
University of Iowa Hosptials and Clinics - Iowa City, Iowa 1,050.4 miles Myrl Holida PA 319-356-2007 myrl-holida@uiowa.edu
University of Iowa Hosptials and Clinics - Iowa City, Iowa 1,050.4 miles Myrl Holida PA 319-356-2007 myrl-holida@uiowa.edu
University of Iowa Hosptials and Clinics - Iowa City, Iowa 1,050.4 miles Myrl Holida PA 319-356-2007 myrl-holida@uiowa.edu
UAB Medicine - Birmingham, Alabama 1,051.7 miles Eric L Wallace MD 205-975-9676 ericlwallace@uab.edu
UAB Medicine - Birmingham, Alabama 1,051.7 miles Eric L Wallace MD 205-975-9676 ericlwallace@uab.edu
UAB Medicine - Birmingham, Alabama 1,051.7 miles Eric L Wallace MD 205-975-9676 ericlwallace@uab.edu
UAB Medicine - Birmingham, Alabama 1,051.7 miles Eric L Wallace MD 205-975-9676 ericlwallace@uab.edu
UAB Medicine - Birmingham, Alabama 1,051.7 miles Eric L Wallace MD 205-975-9676 ericlwallace@uab.edu
UAB Medicine - Birmingham, Alabama 1,051.7 miles Eric L Wallace MD 205-975-9676 ericlwallace@uab.edu
Regents of the University of Minnesota - Minneapolis, Minnesota 1,120.0 miles Chester Whitley 612-625-7422 whitley@umn.edu
Regents of the University of Minnesota - Minneapolis, Minnesota 1,120.0 miles Chester Whitley 612-625-7422 whitley@umn.edu
Regents of the University of Minnesota - Minneapolis, Minnesota 1,120.0 miles Chester Whitley 612-625-7422 whitley@umn.edu
Regents of the University of Minnesota - Minneapolis, Minnesota 1,120.0 miles Chester Whitley 612-625-7422 whitley@umn.edu
Regents of the University of Minnesota - Minneapolis, Minnesota 1,120.0 miles Chester Whitley 612-625-7422 whitley@umn.edu
Institute of Metabolic Disease Baylor Healthcare - Dallas, Texas 1,549.9 miles Raphael Schiffmann MD MHSc 214-820-4533 raphael.schiffmann@baylorhealth.edu
Institute of Metabolic Disease Baylor Healthcare - Dallas, Texas 1,549.9 miles Raphael Schiffmann MD MHSc 214-820-4533 raphael.schiffmann@baylorhealth.edu
Institute of Metabolic Disease Baylor Healthcare - Dallas, Texas 1,549.9 miles Raphael Schiffmann MD MHSc 214-820-4533 raphael.schiffmann@baylorhealth.edu
Institute of Metabolic Disease Baylor Healthcare - Dallas, Texas 1,549.9 miles Raphael Schiffmann MD MHSc 214-820-4533 raphael.schiffmann@baylorhealth.edu
Institute of Metabolic Disease Baylor Healthcare - Dallas, Texas 1,549.9 miles Raphael Schiffmann MD MHSc 214-820-4533 raphael.schiffmann@baylorhealth.edu
Institute of Metabolic Disease Baylor Healthcare - Dallas, Texas 1,549.9 miles Raphael Schiffmann MD MHSc 214-820-4533 raphael.schiffmann@baylorhealth.edu
Eccles Primary Childrens Outpatient Services Building - Salt Lake City, Utah 2,094.1 miles Nicola Longo MD PhD 801-587-3605 nicola.longo@hsc.utah.edu
Eccles Primary Childrens Outpatient Services Building - Salt Lake City, Utah 2,094.1 miles Nicola Longo MD PhD 801-587-3605 nicola.longo@hsc.utah.edu
Eccles Primary Childrens Outpatient Services Building - Salt Lake City, Utah 2,094.1 miles Nicola Longo MD PhD 801-587-3605 nicola.longo@hsc.utah.edu
Eccles Primary Childrens Outpatient Services Building - Salt Lake City, Utah 2,094.1 miles Nicola Longo MD PhD 801-587-3605 nicola.longo@hsc.utah.edu
Eccles Primary Childrens Outpatient Services Building - Salt Lake City, Utah 2,094.1 miles Nicola Longo MD PhD 801-587-3605 nicola.longo@hsc.utah.edu
Eccles Primary Childrens Outpatient Services Building - Salt Lake City, Utah 2,094.1 miles Nicola Longo MD PhD 801-587-3605 nicola.longo@hsc.utah.edu
University of Washington - Seattle, Washington 2,487.2 miles Ron Scott 206-543-0937 crscott@u.washington.edu
University of Washington - Seattle, Washington 2,487.2 miles Ron Scott 206-543-0937 crscott@u.washington.edu
University of Washington - Seattle, Washington 2,487.2 miles Ron Scott 206-543-0937 crscott@u.washington.edu
University of Washington - Seattle, Washington 2,487.2 miles Ron Scott 206-543-0937 crscott@u.washington.edu
University of Washington - Seattle, Washington 2,487.2 miles Ron Scott 206-543-0937 crscott@u.washington.edu
University of Washington - Seattle, Washington 2,487.2 miles Ron Scott 206-543-0937 crscott@u.washington.edu
University of California San Diego - San Diego, California 2,581.5 miles Bruce Barshop 619-543-5237 bbarshop@ucsd.edu
University of California San Diego - San Diego, California 2,581.5 miles Bruce Barshop 619-543-5237 bbarshop@ucsd.edu
University of California San Diego - San Diego, California 2,581.5 miles Bruce Barshop 619-543-5237 bbarshop@ucsd.edu
University of California San Diego - San Diego, California 2,581.5 miles Bruce Barshop 619-543-5237 bbarshop@ucsd.edu
University of California San Diego - San Diego, California 2,581.5 miles Bruce Barshop 619-543-5237 bbarshop@ucsd.edu
University of California Irvine Center - Orange, California 2,585.0 miles Virginia Kimonis 714-456-5791 vkimonis@uci.edu
University of California Irvine Center - Orange, California 2,585.0 miles Virginia Kimonis 714-456-5791 vkimonis@uci.edu
University of California Irvine Center - Orange, California 2,585.0 miles Virginia Kimonis 714-456-5791 vkimonis@uci.edu
University of California Los Angeles - Los Angeles, California 2,604.8 miles Anjay Rastogi 310-954-2692 drrastogioffice@mednet.ucla.edu
University of California Los Angeles - Los Angeles, California 2,604.8 miles Anjay Rastogi 310-954-2692 drrastogioffice@mednet.ucla.edu
University of California Los Angeles - Los Angeles, California 2,604.8 miles Anjay Rastogi 310-954-2692 drrastogioffice@mednet.ucla.edu
University of California Davis Health System - Sacramento, California 2,628.4 miles Madelena Martin 916-703-0203 bcgmartin@ucdavis.edu
University of California Davis Health System - Sacramento, California 2,628.4 miles Madelena Martin 916-703-0203 bcgmartin@ucdavis.edu
University of California Davis Health System - Sacramento, California 2,628.4 miles Madelena Martin 916-703-0203 bcgmartin@ucdavis.edu
University of California Davis Health System - Sacramento, California 2,628.4 miles Madelena Martin 916-703-0203 bcgmartin@ucdavis.edu

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