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 |
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.
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.
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.
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