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



Status:Recruiting
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:18 - 60
Updated:3/30/2019
Start Date:June 2016
End Date:June 2019
Contact:Raul Chertkoff, MD
Email:raul@protalix.com
Phone:+972-4-9028100

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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

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.


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
We found this trial at
25
sites
8701 W Watertown Plank Rd
Milwaukee, Wisconsin
(414) 955-8296
Principal Investigator: William Rhead
Phone: 414-266-3345
Medical College of Wisconsin The Medical College (MCW) of Wisconsin is a major national research...
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1648 Pierce Dr NE
Atlanta, Georgia 30322
(404) 727-5640
Principal Investigator: William Wilcox, MD
Phone: 404-727-5624
Emory University School of Medicine Emory University School of Medicine has 2,359 full- and part-time...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Amel Karaa
Phone: 617-726-1561
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: Robert Hopkin, MD
Phone: 513-636-4760
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Los Angeles, California 90095
310-825-4321
Principal Investigator: Anjay Rastogi, MD
Phone: 310-954-2692
University of California at Los Angeles The University of California, Los Angeles (UCLA) is an...
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920 Madison Ave
Memphis, Tennessee 38163
Principal Investigator: John L. Jefferies, MD
Phone: 901-448-5750
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Amy C. Yang, MD
Phone: 503-418-2385
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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1876 5th Avenue South
Birmingham, Alabama 35233
Principal Investigator: Eric L Wallace, MD
Phone: 205-975-9676
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Joel Charrow, MD
Phone: 773-880-4462
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Dallas, Texas 75226
Principal Investigator: Raphael Schiffmann, MD, MHSc
Phone: 214-820-4533
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Marie McDonald
Phone: 919-681-1982
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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Fairfax, Virginia 22031
Principal Investigator: Ozlem Goker-Alpan, MD
Phone: 571-308-1900
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Gainesville, Florida 32608
Principal Investigator: Maegawa Gustavo, MD, PhD
Phone: 352-294-5559
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Grand Rapids, Michigan 49525
Principal Investigator: Kahn Nedd, MD
Phone: 616-954-0600
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Iowa City, Iowa 52242
Principal Investigator: Myrl Holida, PA
Phone: 319-356-2007
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1 Children's Way
Little Rock, Arkansas 72202
(501) 364-1100
Principal Investigator: Thomas Burrow, MD
Phone: 501-364-2966
Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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Minneapolis, Minnesota 55455
Principal Investigator: Chester Whitley
Phone: 612-625-7422
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Morgantown, West Virginia 26506
(304) 293-0111
Principal Investigator: Tarachandra Narumanchi, MD
Phone: 304-293-7332
West Virginia University West Virginia University, founded in 1867, has a long and rich history...
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New York, New York 10021
Principal Investigator: Heather Lau, MD
Phone: 212-263-8344
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101 The City Drive South
Orange, California 92868
Principal Investigator: Virginia Kimonis
Phone: 714-456-5791
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1115 Avenida de Mayo
Pergamino, Buenos Aires
Principal Investigator: Norberto R Antongiovanni, MD
Phone: +54 2477433928
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Kristin E. Lindstrom, MD
Phone: 602-933-4363
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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3414 Fifth Avenue
Pittsburgh, Pennsylvania 15213
Principal Investigator: Gerard Vockley, Md, PhD
Phone: 412-692-7746
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Salt Lake City, Utah 84113
Principal Investigator: Nicola Longo, MD, PhD
Phone: 801-587-3605
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San Diego, California 92093
Principal Investigator: Bruce Barshop
Phone: 619-543-5237
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