Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients



Status:Enrolling by invitation
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:12/29/2018
Start Date:November 6, 2018
End Date:January 2022

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Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease

The objective of PB-102-F51 is to evaluate the long-term safety, tolerability, and efficacy
of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry
patients who have successfully completed PB-102-F50.

This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase
alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of
treatment will be up to 36 months or until pegunigalsidase alfa is available to the patient
at the discretion of the Sponsor. Interim analyses may be performed for administrative
purposes during the conduct of the study.

Inclusion Criteria:

1. Completion of study PB-102-F50.

2. The patient signs informed consent.

3. Female patients and male patients whose co-partners are of child-bearing potential
agree to use a medically accepted, highly effective method of contraception. These
include combined (estrogen- and progestogen-containing) hormonal contraception
associated with inhibition of ovulation (oral, intravaginal, or transdermal),
progestogen-only hormonal contraception associated with inhibition of ovulation (oral,
injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing
system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence.

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
8
sites
Fairfax, Virginia 22031
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1648 Pierce Dr NE
Atlanta, Georgia 30322
(404) 727-5640
Emory University School of Medicine Emory University School of Medicine has 2,359 full- and part-time...
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Atlanta, GA
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1876 5th Avenue South
Birmingham, Alabama 35233
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Birmingham, AL
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Dallas, TX
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Edegem, 2650
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Edegem,
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Grand Rapids, Michigan 49525
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Grand Rapids, MI
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Iowa City, IA
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Salt Lake City, Utah 84132
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Salt Lake City, UT
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