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

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ClinicalTrials.gov Identifier: NCT03566017
Recruitment Status : Enrolling by invitation
First Posted : June 21, 2018
Last Update Posted : October 24, 2018
Sponsor:
Information provided by (Responsible Party):
Protalix

Brief Summary:
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.

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

Detailed Description:
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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 110 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 Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
Actual Study Start Date : September 20, 2018
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 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: Through study completion, 4 years ]
    CTCAE v4.03


Secondary Outcome Measures :
  1. Kidney function [ Time Frame: Once a month for the first 12 months, then every 3 months until the end of the study, 4 years ]
    Estimated glomerular filtration rate (eGFRCKD-EPI)

  2. Cardiac assessment [ Time Frame: Every 12 months up to the end of the study, 4 years ]
    Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)

  3. Biomarkers for Fabry disease [ Time Frame: Every 3 months the first 12 months and every 6 months up to the end of the study, 4 years ]
    Plasma Gb3, plasma Lyso-Gb3, and urine Lyso-Gb3

  4. Record of pain medication use [ Time Frame: Every two weeks for 4 years ]
    Frequency of pain medication use, or pre-infusion medication

  5. Kidney function [ Time Frame: Every 3 months the first 12 months and every 6 months up to the end of the study, 4 years ]
    Protein/Creatinine ratio, spot urine test (UPCR)

  6. Pain assessment [ Time Frame: Every 6 months up to the end of the study, 4 years ]
    short form Brief Pain Inventory (BPI)

  7. Symptom assessment [ Time Frame: Every 6 months up to the end of the study, 4 years ]
    Mainz Severity Score Index (MSSI)

  8. Quality of life assessment [ Time Frame: Every 6 months up to the end of the study, 4 years ]
    quality of life (EQ-5D-5L)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT03566017


  Show 20 Study Locations
Sponsors and Collaborators
Protalix

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT03566017     History of Changes
Other Study ID Numbers: PB-102-F60
First Posted: June 21, 2018    Key Record Dates
Last Update Posted: October 24, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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:
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