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

Sponsor
Protalix (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT03566017
Collaborator
(none)
110
Enrollment
29
Locations
1
Arm
98.4
Anticipated Duration (Months)
3.8
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

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-F03, PB-102-F20 or PB-102-F30.

Condition or Disease Intervention/Treatment Phase
  • 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 completed studies PB-102-F20, PB-102-F30 or completed at least 36 months in Study PB-102-F03). 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 60 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 Design

Study Type:
Interventional
Anticipated Enrollment :
110 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
Open label extension studyOpen 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 :
Sep 20, 2018
Anticipated Primary Completion Date :
Oct 1, 2026
Anticipated Study Completion Date :
Dec 1, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: Experimental open label

pegunigalsidase alfa

Biological: pegunigalsidase alfa
Recombinant human alpha galactosidase A
Other Names:
  • PRX-102

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Evaluation of treatment-related adverse events [Through study completion, 5 years]

      CTCAE v4.03

    Secondary Outcome Measures

    1. Kidney function [At 6, 14, 28, 40, 54, 80, 106, 132, 158, 184, 210, 236 and 262 weeks]

      Estimated glomerular filtration rate (eGFRCKD-EPI)

    2. Cardiac assessment [Every 24 months up to the end of the study, 5 years]

      Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)

    3. Biomarkers for Fabry disease [Every 12 months to the end of the study, 5 years. For patients from PB-102-F20 at 3 and 6 months.]

      plasma Lyso-Gb3 and Gb3

    4. Record of pain medication use [Every two weeks for 5 years]

      Frequency of pain medication use, or pre-infusion medication

    5. Kidney function [Every 12 months to the end of the study, 5 years. For patients from PB-102-F20 at 3 and 6 months.]

      Protein/Creatinine ratio, spot urine test (UPCR)

    6. Pain assessment [Every 6 months up to the end of the study, 5 years]

      short form Brief Pain Inventory (BPI)

    7. Symptom assessment [Every 12 months up to the end of the study, 5 years]

      Mainz Severity Score Index (MSSI)

    8. Quality of life assessment [Every 6 months up to the end of the study, 5 years]

      quality of life (EQ-5D-5L)

    Eligibility Criteria

    Criteria

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

    • Completion of study PB-102-F20, PB-102-F03, 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 UAB Medicine Birmingham Alabama United States 35233
    2 Phoenix Children's Hospital Phoenix Arizona United States 85016
    3 University of California San Diego La Jolla California United States 92037
    4 University of California Irvine Center Orange California United States 92868
    5 University of Florida Gainesville Florida United States 32610
    6 Emory University School of Medicine Atlanta Georgia United States 30322
    7 University of Iowa Hospitals and Clinics Iowa City Iowa United States 52242
    8 Infusion Associates Grand Rapids Michigan United States 49525
    9 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    10 University of Pittsburgh Pittsburgh Pennsylvania United States 15224
    11 Renal Disease Research Institute, LLC - Dallas Dallas Texas United States 75235
    12 Eccles Primary Children's Outpatient Services Building Salt Lake City Utah United States 84132
    13 O+O Alpan LLC Fairfax Virginia United States 22030
    14 Medical College of Wisconsin Milwaukee Wisconsin United States 53226
    15 Royal Melbourne Hospital Parkville Victoria Australia 3050
    16 Capital District Health Authority Halifax Nova Scotia Canada B3H 1V8
    17 Vseobecna fakultni nemocnice v Praze Prague Czech Republic Czechia 12808
    18 Turku University Central Hospital Turku Finland FI-20521
    19 Hospital Raymond-Poincaré Garches France 92380
    20 Semmelweis Egyetem Budapest Hungary 1083
    21 Azienda Ospedaliera Universitaria "Federico II" Napoli Via Pansini Italy 80131
    22 Academisch Medisch Centrum Amsterdam Netherlands 1105 AZ
    23 Haukeland University Hospital Klinisk Forskningspost Bergen Norway 5021
    24 General Hospital Slovenj Gradec Slovenj Gradec Slovenia 2380
    25 Hospital de Dia Quiron Zaragoza Zaragoza Spain 50012
    26 University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital Edgbaston Birmingham United Kingdom B152TH
    27 Salford Royal Salford Greater Manchester United Kingdom M6 8HD
    28 Addenbrooke's Hospital Cambridge United Kingdom CB2 0QQ
    29 The Royal Free Hospital London United Kingdom NW3 2QG

    Sponsors and Collaborators

    • Protalix

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Protalix
    ClinicalTrials.gov Identifier:
    NCT03566017
    Other Study ID Numbers:
    • PB-102-F60
    First Posted:
    Jun 21, 2018
    Last Update Posted:
    Feb 21, 2022
    Last Verified:
    Feb 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    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

    Study Results

    No Results Posted as of Feb 21, 2022