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

This study is currently recruiting participants.
Verified June 2017 by Protalix
Sponsor:
ClinicalTrials.gov Identifier:
NCT03018730
First Posted: January 12, 2017
Last Update Posted: June 23, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Protalix
  Purpose
This is an open label switch over study to assess the safety and efficacy of PRX-102. 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 Intervention Phase
Fabry Disease Biological: PRX-102 Phase 3

Study Type: Interventional
Study Design: 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)

Resource links provided by NLM:


Further study details as provided by Protalix:

Primary Outcome Measures:
  • Treatment-emergent anti-PRX-102 antibodies [ Time Frame: 12 months ]

Secondary Outcome Measures:
  • Mean annualised change in eGFR [ Time Frame: Every 2 weeks for 12 months ]
    Mean annualised change in eGFR

  • 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)

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

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

Estimated Enrollment: 22
Actual Study Start Date: February 23, 2017
Estimated Study Completion Date: September 2018
Estimated Primary Completion Date: June 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PRX-102
PRX-102 infusion every 2 weeks
Biological: PRX-102
PRX-102 1 mg/kg every 2 weeks
Other Names:
  • pegunigalsidase alfa
  • Recombinant human alpha galactosidase-A

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

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


Contacts
Contact: Mali Szlaifer, MS 972(0)4-902-8100 ext 180 malis@protalix.com

Locations
Australia, Victoria
Royal Melbourne Hospital Recruiting
Parkville, Victoria, Australia, 3050
Contact: Kathleen Nicholls, MD    61393427000    kathy.nicholls@mh.org.au   
Principal Investigator: Kathleen Nicholls, MD         
Canada, Nova Scotia
Capital Health Recruiting
Halifax, Nova Scotia, Canada, B3H 1V8
Contact: Michael L West, MD    9024734023    mlwest@dal.ca   
Principal Investigator: Michael L West, MD         
Czechia
Vseobecna fakultni nemocnice v Praze Recruiting
Prague, Czechia
Contact: Ales Linhart, MD    420224962605    ales.linhart@lf1.cuni.cz   
Principal Investigator: Ales Linhart, MD         
Germany
Universitaetsklinikum Wuerzburg Not yet recruiting
Wurzburg, Germany
Contact: Christoph Wanner, MD    49931020139030    wanner_c@klinik.uni-wuerzburg.de   
Principal Investigator: Christoph Wanner, MD         
Netherlands
Academisch Medisch Centrum Not yet recruiting
Amsterdam, Netherlands
Contact: CEM Hollak, MD    31205666071    c.e.hollak@amc.uva.nl   
Principal Investigator: CEM Hollak, MD         
Norway
Helse Bergen HF Haukeland Universitetssykehus Recruiting
Bergen, Norway
Contact: Camilla Tøndel, MD    4755973859    camilla.tondel@helse-bergen.no   
Principal Investigator: Camilla Tøndel, MD         
Spain
Hospital de Dia Quiron Zaragoza Not yet recruiting
Zaragoza, Spain, 50012
Contact: Beatriz Escuder    34 976 765 500    bea.escuderazuara@gmail.com   
Principal Investigator: Pilar Giraldo, MD         
United Kingdom
Addenbrooke's Hospital Not yet recruiting
Cambridge, United Kingdom, CB2 0QQ
Contact: Patrick Deegan, MD    441223245151    patrick.deegan@addenbrookes.nhs.uk   
Principal Investigator: Patrick Deegan, MD         
The Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Derralynn Hughes, MD    44 207 472 6588    rmgvdah@ucl.ac.uk   
Principal Investigator: Derralynn Hughes, MD         
Salford Royal NHS Foundation Trust Not yet recruiting
Salford, United Kingdom, M6 8HD
Contact: Ana Jovanovic, MD    441612064365    ana.jovanovic@srft.nhs.uk   
Principal Investigator: Ana Jovanovic, MD         
Sponsors and Collaborators
Protalix
Investigators
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 Submitted: January 9, 2017
First Posted: January 12, 2017
Last Update Posted: June 23, 2017
Last Verified: June 2017
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

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