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Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function (BALANCE)

This study is currently recruiting participants.
Verified September 2017 by Protalix
Sponsor:
ClinicalTrials.gov Identifier:
NCT02795676
First Posted: June 10, 2016
Last Update Posted: October 2, 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 a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with 1mg/kg agalsidase beta or to treatment with 1 mg/kg of PRX-102. The identity of the enzyme will be blinded to the patient and the investigator. Patients will receive intravenous infusions every two weeks. Patients will be randomized in a 2:1 ratio of PRX-102 to agalsidase beta. Randomization will be stratified by urinary protein to creatinine ratio (UPCR) of < or ≥ 1 g/g by spot urine sample. No more than 50% of the patients will be female.

Condition Intervention Phase
Fabry Disease Biological: PRX-102 (pegunigalsidase alfa) Biological: agalsidase beta Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta

Resource links provided by NLM:


Further study details as provided by Protalix:

Primary Outcome Measures:
  • eGFR Slope [ Time Frame: Every month for 2 years ]
    eGFR Slope


Secondary Outcome Measures:
  • Left Ventricular Mass Index (g/m2) by MRI [ Time Frame: Every 12 months for 2 years ]
  • Plasma Lyso-Gb3 [ Time Frame: Every 3 months for 2 years ]
  • Plasma Gb3 [ Time Frame: Every 3 months for 2 years ]
  • Urine Lyso-GB3 [ Time Frame: Every 6 weeks for 2 years ]
  • Protein/creatinine ratio [ Time Frame: Every 4 months for 24 months ]
  • Frequency of pain medication use [ Time Frame: Every 2 weeks for 2 years ]
  • Exercise tolerance (Stress Test) [ Time Frame: Every year for 2 years ]
  • Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 6 months for 2 years ]
  • Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 2 years ]
  • Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 2 years ]

Other Outcome Measures:
  • PRX-102 pharmacokinetics [ Time Frame: Every 6 months for 2 years ]
    PRX-102 pharmacokinetics parameters

  • Anti-drug IgG antibodies [ Time Frame: Every 2 weeks for 1 month, then every month for the first 6 months and every 3 month until the end of the study ]

Estimated Enrollment: 78
Study Start Date: June 2016
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: March 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PRX-102 (pegunigalsidase alfa)
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
Active Comparator: agalsidase beta
agalsidase beta infusion every 2 weeks
Biological: agalsidase beta
agalsidase beta 1 mg/kg every 2 weeks
Other Name: Fabrazyme

  Eligibility

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
Criteria

Inclusion Criteria:

  • Symptomatic adult Fabry disease patients, age 18-60 years

    1. Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels and one or more of the characteristic features of Fabry disease

      i. neuropathic pain

      ii. cornea verticillata

      iii. clustered angiokeratoma

    2. Females:

      a. historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:

      i. neuropathic pain

      ii. cornea verticillata

      iii. clustered angiokeratoma

      b. or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease

      i. neuropathic pain

      ii. cornea verticillata

      iii. clustered angiokeratoma

  • Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
  • Linear negative slope of eGFR based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit) of ≥ 2 mL/min/1.73 m²/year
  • Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months.
  • Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.

Exclusion Criteria:

  • History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
  • Known non-pathogenic Fabry mutations
  • History of renal dialysis or transplantation
  • 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)
  • Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
  • Patient with a screening eGFR value between 91-120 mL/min/1.73 m², having an historical eGFR value higher than 120 mL/min/1.73 m² (during 9 to 18 months before screening)
  • Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
  • Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
  • Congestive heart failure NYHA Class IV
  • Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
  • Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of pre-medication
  • Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
  • 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): NCT02795676


Contacts
Contact: Raul Chertkoff, MD +972-4-9028100 raul@protalix.com
Contact: Mali Szlaifer, MS malis@protalix.com

  Show 38 Study Locations
Sponsors and Collaborators
Protalix
Investigators
Study Director: Raul Chertkoff, MD Protalix Ltd
  More Information

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT02795676     History of Changes
Other Study ID Numbers: PB-102-F20
First Submitted: June 2, 2016
First Posted: June 10, 2016
Last Update Posted: October 2, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Protalix:
Glomerular filtration rate
Proteinuria
PRX-102
pegunigalsidase alfa
Fabry Disease

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