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Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

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ClinicalTrials.gov Identifier: NCT03018730
Recruitment Status : Recruiting
First Posted : January 12, 2017
Last Update Posted : January 17, 2018
Information provided by (Responsible Party):

Study Description
Brief Summary:
This is an open label switch over study to assess the safety and efficacy of PRX-102 (pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months. Patients will be screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, the patient will be enrolled and switched from their agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25% of treated patients will be female.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: PRX-102 (pegunigalsidase alfa) Phase 3

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 22 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Actual Study Start Date : February 23, 2017
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : September 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Throughout the 12 months study ]

Secondary Outcome Measures :
  1. Mean annualised change in eGFR [ Time Frame: Every 4 weeks for 12 months ]
    Mean annualised change in eGFR

  2. Left Ventricular Mass Index (g/m2) preferably by MRI [ Time Frame: Every 6 months for 12 months ]
    Left Ventricular Mass Index (g/m2) preferably by MRI (ECG can be used as an alternative)

  3. Plasma Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
  4. Plasma Gb3 [ Time Frame: Every 3 months for 12 months ]
  5. Urine Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
  6. Protein/Creatinine ratio [ Time Frame: Every 3 months for 12 months ]
    Protein/Creatinine ratio spot urine test

  7. Frequency of pain medication use [ Time Frame: Every 2 weeks for 12 months ]
  8. Exercise tolerance (Stress Test) [ Time Frame: Every 6 months for 12 months ]
  9. Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 3 months for 12 months ]
  10. Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 12 months ]
  11. Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 12 months ]

Eligibility Criteria

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

Inclusion Criteria:

  1. Age: 18-60 years
  2. A documented diagnosis of Fabry disease
  3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal according to laboratory range and one or more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata iii. Clustered angiokeratoma
  4. Females: historical genetic test results consistent with Fabry mutations, or in the case of novel mutations a first degree male relative with Fabry disease, and one or more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata iii. Clustered angiokeratoma
  5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months
  6. eGFR ≥ 40 ml/min/1.73 m2 by CKD-EPI equation
  7. Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years
  8. 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

Exclusion Criteria:

  1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa
  2. History of renal dialysis or transplantation
  3. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
  4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
  5. Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
  6. Known history of hypersensitivity to Gadolinium contrast agent
  7. Females who are pregnant, planning to become pregnant during the study, or are breast feeding
  8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening
  9. Congestive heart failure NYHA Class IV
  10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening
  11. 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
Contacts and Locations

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): NCT03018730

Contact: Raul Chertkoff, MD +972-54-2228472 raul@protalix.com

Australia, Victoria
Royal Melbourne Hospital Recruiting
Parkville, Victoria, Australia, 3050
Contact: Kathleen Nicholls, MD    61393427000    kathy.nicholls@mh.org.au   
Contact: Donna North    +61 393424219    donna.north@mh.org.au   
Principal Investigator: Kathleen Nicholls, MD         
Canada, Nova Scotia
Capital Health Recruiting
Halifax, Nova Scotia, Canada, B3H 1V8
Contact: Michael West, MD    +1 902 473 4023    mlwest@dal.ca   
Contact: Dawn Menon    +1 9024731156    Dawn.Menon@nshealth.ca   
Principal Investigator: Michael West, MD         
Vseobecna fakultni nemocnice v Praze Recruiting
Prague, Czechia
Contact: Ales Linhart, MD    420224962605    alinh@lf1.cuni.cz   
Contact: Zuzana Kristofova    420 224 962 605    zuzana.kristofova@vfn.cz   
Principal Investigator: Ales Linhart, MD         
Universitaetsklinikum Wuerzburg Not yet recruiting
Wurzburg, Germany
Contact: Christoph Wanner, MD    49 931 20139030    wanner_c@klinik.uni-wuerzburg.de   
Principal Investigator: Christoph Wanner, MD         
Academisch Medisch Centrum Recruiting
Amsterdam, Netherlands
Contact: Mirjam Langeveld, MD    31205666071    m.langeveld@amc.nl   
Contact: Shirley Klein    31205665972    shirley.klein@amc.uva.nl   
Principal Investigator: Mirjam Langeveld, MD         
Helse Bergen HF Haukeland Universitetssykehus Recruiting
Bergen, Norway
Contact: Camilla Tøndel, MD    +47 55975962    camilla.tondel@helse-bergen.no   
Contact: Jorunn Syltoy    +47 55971490    jorunn.johanne.jacobsen.syltoy@helse-bergen.no   
Principal Investigator: Camilla Tøndel, MD         
General Hospital Slovenj Gradec Recruiting
Slovenj Gradec, Slovenia, SI-2380
Contact: Bojan Vujkovac, MD    00386 2 8823 708    bojan.vujkovac@guest.arnes.si   
Principal Investigator: Bojan Vujkovac, MD         
Hospital de Dia Quiron Zaragoza Recruiting
Zaragoza, Spain, 50012
Contact: Pilar Giraldo, MD    34670285339    giraldocastellano@gmail.com   
Contact: Beatriz Escuder    34676605758    bea.escuderazuara@gmail.com   
Principal Investigator: Pilar Giraldo, MD         
United Kingdom
Institute of Metabolism and Systems Research Not yet recruiting
Edgbaston, Birmingham, United Kingdom, B15 2UR
Contact: Tarekegn G Hiwot, MD    +44 (0)121 371 6982    t.g.hiwot@bham.ac.uk   
Contact: Tarekegn G Hiwot, MD    +44 (0)121 371 6982    tarekegn.hiwot@uhb.nhs.uk   
Principal Investigator: Tarekegn G Hiwot, MD         
Addenbrooke's Hospital Recruiting
Cambridge, United Kingdom, CB2 0QQ
Contact: Patrick Deegan, MD    441223245151    patrick.deegan@addenbrookes.nhs.uk   
Contact: Lisa Morris-Bacon    441223 274634 ext 4634    lisa.morris-bacon@addenbrookes.nhs.uk   
Principal Investigator: Patrick Deegan, MD         
The Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Derralynn Hughes, MD    4402077940500 ext 22496    rmgvdah@ucl.ac.uk   
Contact: Allison Warwick    44 0207 7940500 ext 22488    allison.warwick@nhs.net   
Principal Investigator: Derralynn Hughes, MD         
Salford Royal NHS Foundation Trust Recruiting
Salford, United Kingdom, M6 8HD
Contact: Ana Jovanovic, MD    +441612064365    ana.jovanovic@srft.nhs.uk   
Contact: Marie Meehan    +441612064192    Marie.Meehan@srft.nhs.uk   
Principal Investigator: Ana Jovanovic, MD         
Sponsors and Collaborators
Study Director: Raul Chertkoff, MD Protalix Ltd
More Information

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT03018730     History of Changes
Other Study ID Numbers: PB-102-F30
First Posted: January 12, 2017    Key Record Dates
Last Update Posted: January 17, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Keywords provided by Protalix:
Fabry disease
Enzyme-Replacement Therapy
pegunigalsidase alfa
alpha galactosidase-A

Additional relevant MeSH terms:
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