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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03614234
Recruitment Status : Active, not recruiting
First Posted : August 3, 2018
Last Update Posted : April 21, 2023
Sponsor:
Information provided by (Responsible Party):
Chiesi Farmaceutici S.p.A.

Study Description
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Brief Summary:
The objective of CLI-06657AA1-03 (formerly 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 Drug: 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 until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: N/A
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 13, 2018
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


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


Outcome Measures
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Primary Outcome Measures :
  1. Evaluation of treatment-related adverse events [ Time Frame: Throughout the study, 364 weeks ]
    CTCAE v4.03


Secondary Outcome Measures :
  1. Kidney function 1 [ Time Frame: Every 6 months throughout the duration of the study, 364 weeks ]
    Estimated glomerular filtration rate (eGFRCKD-EPI)

  2. Cardiac assessment [ Time Frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 ]
    Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test

  3. Biomarkers for Fabry disease [ Time Frame: Every 6 months throughout the duration of the study, 364 weeks ]
    Plasma Lyso-Gb3 and Gb3

  4. Kidney function 2 [ Time Frame: Every 6 months throughout the duration of the study, 364 weeks ]
    Protein/Creatinine ratio (UPCR), spot urine test

  5. Clinical assessment [ Time Frame: Every four weeks throughout the duration of the study, 364 weeks ]
    Record of pain medication and pre-medication use

  6. Pain assessment [ Time Frame: Every 6 months throughout the duration of the study, 364 weeks ]
    Short form Brief Pain Inventory (BPI)

  7. Symptom assessment [ Time Frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 ]
    Mainz Severity Score Index (MSSI)

  8. Quality of life assessment [ Time Frame: Every 6 months throughout the duration of the study, 364 weeks ]
    Quality of life (EQ-5D-5L)


Eligibility Criteria
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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, effective contraception method. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.

Exclusion Criteria:

Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.

Contacts and Locations
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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
Renal Disease Research Institute, LLC
Dallas, Texas, United States, 75235
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
Czechia
Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
Praha, Czechia, 128 08
Denmark
Medical Endocrinology PE 2132, Rigshospitalet
Copenhagen, Denmark, 2100
Italy
Azienda Ospedaliera Universitaria "Federico II"
Napoli, Via Pansini, Italy, 80131
Norway
Helse Bergen HF Haukeland Universitetssykehus
Bergen, Norway, 5021
United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom, CB2 0QQ
The Royal Free Hospital
London, United Kingdom
Sponsors and Collaborators
Chiesi Farmaceutici S.p.A.
More Information
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Responsible Party: Chiesi Farmaceutici S.p.A.
ClinicalTrials.gov Identifier: NCT03614234    
Other Study ID Numbers: CLI-06657AA1-03
First Posted: August 3, 2018    Key Record Dates
Last Update Posted: April 21, 2023
Last Verified: April 2023

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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 Chiesi Farmaceutici S.p.A.:
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