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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. (see Contacts and Locations)
Verified January 2017 by Protalix
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT02795676
First received: June 2, 2016
Last updated: January 9, 2017
Last verified: January 2017
  Purpose
This is a randomized, double blind, active control study of PRX-102 in Fabry disease patients with impaired renal function. Patients treated for at least 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 either agalsidase beta at the same dose 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.

Condition Intervention Phase
Fabry Disease
Biological: PRX-102
Biological: Agalsidase beta
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (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 ]
    Annualized change in eGFR


Secondary Outcome Measures:
  • Left Ventricular Mass Index (g/m2) by MRI [ Time Frame: Every 12 months for 2 years ]
  • Body weight [ Time Frame: Every 2 weeks for 24 months ]
  • 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 ]

Other Outcome Measures:
  • Area under the curve [ Time Frame: Every 6 months for 2 years ]
    PRX-102 pharmacokinetics to evaluate comparative AUCs

  • Anti-drug antibodies [ Time Frame: Every 4 months for 2 years ]

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
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
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

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

    i. neuropathic pain, ii. cornea verticillata, iii. clustered angiokeratoma

  • Screening eGFR by CKD-EPI equation 40 to 90 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.

Exclusion Criteria:

  • History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
  • 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
  • Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or AR
  • 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
  • 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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02795676

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

  Show 26 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
Study First Received: June 2, 2016
Last Updated: January 9, 2017
Individual Participant Data  
Plan to Share IPD: Undecided

Keywords provided by Protalix:
Glomerular filtration rate
Proteinuria

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

ClinicalTrials.gov processed this record on April 28, 2017