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Extension Study of PRX-102 for up to 60 Months

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: NCT01981720
Recruitment Status : Completed
First Posted : November 11, 2013
Results First Posted : February 8, 2023
Last Update Posted : February 8, 2023
Information provided by (Responsible Party):

Brief Summary:
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: PRX-102 (pegunigalsidase alfa) Phase 1 Phase 2

Detailed Description:
An open-label study to evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients (≥18 years of age). Patients enrolled received 1.0 mg/kg of PRX-102 as an intravenous infusion every 2 weeks (+/- 3 days) for up to 60 months and no less than 36 months.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry Patients
Actual Study Start Date : January 16, 2014
Actual Primary Completion Date : August 26, 2020
Actual Study Completion Date : November 9, 2021

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: PRX-102 (pegunigalsidase alfa)
PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)
Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
  • pegunigalsidase alfa
  • Recombinant human alpha galactosidase-A

Primary Outcome Measures :
  1. Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03 [ Time Frame: Every two weeks up to 60 months ]
    Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment.

Other Outcome Measures:
  1. Plasma Lyso-Gb3 Concentration [ Time Frame: Baseline and month 60 ]
    Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker, which was measured at Baseline, every 3 months up to 24 months and then every 6 months up to the end of the study. Baseline and Month 60 and change from Baseline reported.

  2. Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline and Month 60 ]
    eGFR was calculated based on the serum creatinine values according to the CKD-EPI equation. The absolute change in eGFR from baseline measurement at visit 1 to Month 60 was summarized using descriptive statistics. Baseline and month 60 reported.

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

Inclusion Criteria:

  • Completion of study PB-102-F02
  • The patient signs informed consent
  • Female patients and male patients whose co-partners were 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 throughout the duration of the study and for 3 months after termination of treatment.

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 the patient's 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): NCT01981720

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United States, Florida
University of Florida
Gainesville, Florida, United States, 32611
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
United States, Texas
Institute of Metabolic Disease
Dallas, Texas, United States, 75226
United States, Virginia
O & O Alpan
Fairfax, Virginia, United States, 22030
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Asunción, Paraguay
Hospital de Dia Quiron Zaragoza
Zaragoza, Spain, 50012
United Kingdom
The Royal Free Hospital
London, United Kingdom, NW3 2QG
Sponsors and Collaborators
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Study Director: Raul Chertkoff, MD Protalix Ltd.
  Study Documents (Full-Text)

Documents provided by Protalix:
Study Protocol  [PDF] January 5, 2016
Statistical Analysis Plan  [PDF] June 2, 2021

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Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01981720    
Other Study ID Numbers: PB-102-F03
First Posted: November 11, 2013    Key Record Dates
Results First Posted: February 8, 2023
Last Update Posted: February 8, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Keywords provided by Protalix:
pegunigalsidase alfa
Fabry Disease
Additional relevant MeSH terms:
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Fabry Disease
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders