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

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ClinicalTrials.gov Identifier: NCT03614234
Recruitment Status : Enrolling by invitation
First Posted : August 3, 2018
Last Update Posted : December 27, 2018
Sponsor:
Information provided by (Responsible Party):
Protalix

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: pegunigalsidase alfa Phase 3

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Open-label extension study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : November 6, 2018
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : January 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Experimental open label
Pegunigalsidase alfa
Biological: pegunigalsidase alfa
Recombinant human alpha galactosidase A
Other Name: PRX-102




Primary Outcome Measures :
  1. Evaluation of treatment-related adverse events [ Time Frame: Throughout the study, 156 weeks ]
    CTCAE v4.03


Secondary Outcome Measures :
  1. Kidney function [ Time Frame: At 56 and108 weeks and at the end of the study, 156 weeks ]
    Estimated glomerular filtration rate (eGFRCKD-EPI)

  2. Cardiac assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]
    Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test

  3. Biomarkers for Fabry disease [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]
    Plasma Lyso-Gb3

  4. Clinical assessment [ Time Frame: Every four weeks throughout the duration of the study, 156 weeks ]
    Record of pain medication use

  5. Kidney function [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]
    Protein/Creatinine ratio (UPCR), spot urine test

  6. Pain assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]
    Short form Brief Pain Inventory (BPI)

  7. Symptom assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]
    Mainz Severity Score Index (MSSI)

  8. Quality of life assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]
    Quality of life (EQ-5D-5L)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03614234


Locations
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United States, Alabama
UAB Medicine
Birmingham, Alabama, United States, 35233
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30307
United States, Iowa
University of Iowa Hospitals and Clinica
Iowa City, Iowa, United States, 52242
United States, Michigan
Infusion Associates
Grand Rapids, Michigan, United States, 49525
United States, Texas
Baylor Institute of Metabolic Disease
Dallas, Texas, United States, 75226
United States, Utah
University of Utah Hospitals & Clinics
Salt Lake City, Utah, United States, 84112
United States, Virginia
O & O Alpan
Fairfax, Virginia, United States, 22030
Belgium
UZ Antwerpen
Edegem, Belgium, 2650
Norway
Helse Bergen HF Haukeland Universitetssykehus
Bergen, Norway, 5021
United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom, CB2 0QQ
Sponsors and Collaborators
Protalix

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Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT03614234     History of Changes
Other Study ID Numbers: PB-102-F51
First Posted: August 3, 2018    Key Record Dates
Last Update Posted: December 27, 2018
Last Verified: July 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Protalix:
Glomerular filtration rate
Proteinuria
PRX-102
pegunigalsidase alfa
Fabry disease

Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders