Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease



Status:Enrolling by invitation
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:18 - 60
Updated:11/23/2018
Start Date:September 20, 2018
End Date:September 2022

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Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy
parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in
adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30.

The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy
parameters of 1 mg/kg pegunigalsidase alfa every other week in adult Fabry patients who have
successfully completed studies PB-102-F20 or PB-102-F30. Patients will be enrolled to receive
1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration
of treatment will be no less than 24 months and up to 48 months or until pegunigalsidase alfa
is commercially available to the patient at the discretion of the Sponsor. An interim
analysis may be performed for administrative purposes during the conduct of the study. For
the analysis, available efficacy and safety parameters will be summarized using descriptive
statistics.

Inclusion Criteria:

- Completion of study PB-102-F20 or PB-102-F30

- The patient signs informed consent

- Female patients and male patients whose co-partners are of child-bearing potential
agree to use a medically acceptable method of contraception, not including the rhythm
method. Acceptable methods of contraception include hormonal products, intrauterine
device, or male or female condoms. Contraception should be used for 1 month after
treatment termination.

Exclusion Criteria:

- Presence of any medical, emotional, behavioral or psychological condition that, in the
judgment of the Investigator and/or Medical Director, would interfere with patient
compliance with the requirements of the study.
We found this trial at
11
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Parkville, Victoria
Phone: +61393427000
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1648 Pierce Dr NE
Atlanta, Georgia 30322
(404) 727-5640
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
Phone: 617-726-1561
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Boston, MA
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Phone: 513-636-4760
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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1876 5th Avenue South
Birmingham, Alabama 35233
Phone: 205-975-9676
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Birmingham, AL
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Dallas, Texas 75226
Phone: 214-820-4533
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Dallas, TX
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Fairfax, Virginia 22031
Phone: 571-308-1900
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Fairfax, VA
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Grand Rapids, Michigan 49525
Phone: 616-954-0600
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Grand Rapids, MI
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200 Hawkins Dr,
Iowa City, Iowa 52242
866-452-8507
Phone: 319-356-2007
University of Iowa Hospitals and Clinics University of Iowa Hospitals and Clinics—recognized as one of...
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Iowa City, IA
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101 The City Drive South
Orange, California 92868
Phone: 714-456-5791
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Orange, CA
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Salt Lake City, UT
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