Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 5 for:    Fabrazyme | Recruiting, Active, not recruiting, Enrolling by invitation Studies | Fabry disease
Previous Study | Return to List | Next Study

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

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: NCT02795676
Recruitment Status : Active, not recruiting
First Posted : June 10, 2016
Last Update Posted : October 30, 2019
Sponsor:
Information provided by (Responsible Party):
Protalix

Tracking Information
First Submitted Date  ICMJE June 2, 2016
First Posted Date  ICMJE June 10, 2016
Last Update Posted Date October 30, 2019
Study Start Date  ICMJE June 2016
Estimated Primary Completion Date October 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 28, 2017)
eGFR Slope [ Time Frame: Every month for 2 years ]
eGFR Slope
Original Primary Outcome Measures  ICMJE
 (submitted: June 6, 2016)
eGFR Slope [ Time Frame: Every month for 2 years ]
Annualized change in eGFR
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 28, 2017)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 6, 2016)
  • 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 ]
Current Other Pre-specified Outcome Measures
 (submitted: September 28, 2017)
  • 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 ]
Original Other Pre-specified Outcome Measures
 (submitted: June 6, 2016)
  • 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 ]
 
Descriptive Information
Brief Title  ICMJE Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Fabry Disease
Intervention  ICMJE
  • Biological: PRX-102 (pegunigalsidase alfa)
    PRX-102 1 mg/kg every 2 weeks
    Other Names:
    • pegunigalsidase alfa
    • Recombinant human alpha galactosidase-A
  • Biological: agalsidase beta
    agalsidase beta 1 mg/kg every 2 weeks
    Other Name: Fabrazyme
Study Arms  ICMJE
  • Experimental: PRX-102 (pegunigalsidase alfa)
    PRX-102 infusion every 2 weeks
    Intervention: Biological: PRX-102 (pegunigalsidase alfa)
  • Active Comparator: agalsidase beta
    agalsidase beta infusion every 2 weeks
    Intervention: Biological: agalsidase beta
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: October 25, 2016)
78
Original Estimated Enrollment  ICMJE
 (submitted: June 6, 2016)
69
Estimated Study Completion Date  ICMJE May 2022
Estimated Primary Completion Date October 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Belgium,   Brazil,   Canada,   Czechia,   Finland,   France,   Germany,   Hungary,   Italy,   Netherlands,   Norway,   Paraguay,   Slovenia,   Spain,   Switzerland,   Turkey,   United Kingdom,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number  ICMJE NCT02795676
Other Study ID Numbers  ICMJE PB-102-F20
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Protalix
Study Sponsor  ICMJE Protalix
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Raul Chertkoff, MD Protalix Ltd.
PRS Account Protalix
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP