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

Who is this study for? Patients with Fabry Disease
Status: Completed
Location: See all (28) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 60
Healthy Volunteers: No
View:

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

• 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

• 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.

Locations
United States
Alabama
UAB Medicine
Birmingham
Arizona
Phoenix Children's Hospital
Phoenix
California
University of California Irvine Center
Orange
University of California San Diego
San Diego
Georgia
Emory University School of Medicine
Atlanta
Iowa
University of Iowa Hosptials and Clinics
Iowa City
Massachusetts
Massachusetts General Hospital
Boston
Michigan
Infusion Associates
Grand Rapids
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh
Texas
Institute of Metabolic Disease, Baylor Healthcare
Dallas
Renal Disease Research Institute, LLC - Dallas
Dallas
Utah
Eccles Primary Children's Outpatient Services Building
Salt Lake City
Virginia
O+O Alpan LLC
Fairfax
Wisconsin
Medical College of Wisconsin
Milwaukee
Other Locations
Finland
Turku University Central Hospital
Turku
France
Hôpital Raymond Poincaré
Paris
Hungary
Semmelweis Egyetem
Budapest
Italy
Azienda Ospedaliera Universitaria Federico II
Napoli
Netherlands
Academisch Medisch Centrum
Amsterdam
Norway
Haukeland University Hospital Klinisk Forskningspost
Bergen
Slovenia
General Hospital Slovenj Gradec
Slovenj Gradec
Spain
Hospital de Dia Quiron Zaragoza
Zaragoza
Switzerland
Klinik und Poliklinik für Innere Medizin UniversitätsSpital Zürich
Zürich
United Kingdom
Addenbrooke's Hospital
Cambridge
Institute of Metabolism and Systems Research
Edgbaston
The Royal Free Hospital
London
Salford Royal NHS Foundation Trust
Salford
Time Frame
Start Date: June 2016
Completion Date: July 2022
Participants
Target number of participants: 78
Treatments
Experimental: PRX-102 (pegunigalsidase alfa)
PRX-102 infusion every 2 weeks
Active Comparator: agalsidase beta
agalsidase beta infusion every 2 weeks
Authors
Cole Duncan, Maegawa Gustavo, Pisani Antonio, Albina N Oberärztin, Bernat John, Eugenio E Minetti, Kahn Nedd, Raphael Schiffmann, William Wilcox, Chester Whitley, Thomas Burrow, Heather Lau, Bruce Barshop, Amel Karaa, Gerard Vockley, Madelena Martin, Robert Hopkin, Virginia Kimonis, Joel Charrow, Eric L Wallace, Ozlem Goker-Alpan, Kristin E. Lindstrom, Anjay Rastogi, Amy C. Yang, Marie McDonald, Coy Heldermon, Myrl Holida, Michael L West, Christoph Wanner, Ana Jovanovic, Norberto R Antongiovanni, Robert Giugliani, Maria J Molnar, Charles M Lourenço, Bojan Vujkovac, Raffaele Manna, Kathleen Nicholls, Patrick Deegan, F Ezgu, Derralynn Hughes, William Rhead, Kalkan Sema, Derlis Emilio Gonzales Rodriguez, Nicola Longo, Ron Scott, Mirjam Langeveld, John L. Jefferies, Ales Linhart, Sema Kalkan Ucar, Camilla Tøndel, Tarekegn G Hiwot, Francois Eyskens, Tarachandra Narumanchi, Pilar Giraldo, CEM Hollak, Robin Lachmann
Related Therapeutic Areas
Sponsors
Leads: Protalix

This content was sourced from clinicaltrials.gov