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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02795676
Registration number
NCT02795676
Ethics application status
Date submitted
2/06/2016
Date registered
10/06/2016
Date last updated
13/09/2023
Titles & IDs
Public title
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
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Scientific 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
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Secondary ID [1]
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PB-102-F20
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Universal Trial Number (UTN)
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Trial acronym
BALANCE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - PRX-102 (pegunigalsidase alfa)
Other interventions - agalsidase beta
Experimental: PRX-102 (pegunigalsidase alfa) - PRX-102 infusion every 2 weeks
Active Comparator: agalsidase beta - agalsidase beta infusion every 2 weeks
Other interventions: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other interventions: agalsidase beta
agalsidase beta 1 mg/kg every 2 weeks
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR)
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Assessment method [1]
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The individual annualized mean change (slope) in eGFR (mL/min/1.73 m^2/year) is an estimate of the individual patient's annualized change in eGFR, which is derived from the eGFR assessments over time, for up to 24 months.
The individual annualized mean change (slope) in eGFR is estimated for each patient with at least 4 eGFR observations. For patients with fewer than 4 eGFR observations, the slope will be missing.
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Timepoint [1]
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24 months
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Secondary outcome [1]
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Estimated Glomerular Filtration Rate (eGFR)
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Assessment method [1]
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eGFR was calculated based on measured serum creatinine levels according to the CKD-EPI formula. The median values obtained at baseline and at Month 24 are reported.
The change in eGFR from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
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Timepoint [1]
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Baseline and Month 24
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Secondary outcome [2]
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Plasma Lyso-Gb3
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Assessment method [2]
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Globotriaosylsphingosine (lyso-Gb3) is a Fabry disease-specific biomarker measured in the plasma. The median concentrations obtained at baseline and at Month 24, and the change from baseline to Month 24 in median concentration, are reported.
The change in Lyso-Gb3 from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
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Timepoint [2]
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Baseline and Month 24
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Secondary outcome [3]
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Short Form Brief Pain Inventory (BPI)
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Assessment method [3]
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The Short Form Brief Pain Inventory ( BPI) questioner is self-completed by patients regarding pain severity and interference.
Descriptive statistics summarize the findings for the change from baseline at Week 104 for "Pain at Its Worst in Last 24 Hours".
The severity of various aspects of pain scored on a scale of 0 to 10 ( no pain / pain as bad as you can imagine).
The change in BPI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes.
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Timepoint [3]
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Baseline and Month 24
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Secondary outcome [4]
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Mainz Severity Score Index (MSSI)
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Assessment method [4]
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The Mainz Severity Score Index (MSSI) is an instrument that is specifically designed to measure the severity of Fabry disease signs/symptoms and to monitor the clinical course of the disease. The MSSI is administered by the investigator, and yields scores for general, neurological, cardiovascular, renal, and overall assessments. The overall score range from 0 to 76. An overall score of less than 20 points is considered mild signs and symptoms of Fabry disease, 20 to 40 is considered moderate, and greater than 40 is considered severe.
The change in overall MSSI score from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes.
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Timepoint [4]
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Baseline and Month 24
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Secondary outcome [5]
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Urine Protein/Creatinine Ratio (UPCR)
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Assessment method [5]
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The UPCR provides an estimate of protein excretion in urine, and is used as an indicator of the extent of chronic kidney disease. It was classified into three categories: 1) UPCR = 0.5 gr/gr, 2) 0.5 gr/gr < UPCR < 1 gr/gr, 3) 1 gr/gr = UPCR. The results are presented as the percentage of patients (%) in each category at baseline and Month 24.
There are no statistical analyses for this endpoint.
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Timepoint [5]
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Baseline and Month 24
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Secondary outcome [6]
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Left Ventricular Mass Index With Hypertrophy at Baseline
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Assessment method [6]
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Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients with hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2).
The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
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Timepoint [6]
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Baseline and Month 24
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Secondary outcome [7]
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Left Ventricular Mass Index Without Hypertrophy at Baseline
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Assessment method [7]
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Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients without hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2).
The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
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Timepoint [7]
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Baseline and Month 24
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Eligibility
Key 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.
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Minimum age
18
Years
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Maximum age
60
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/07/2022
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Sample size
Target
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Accrual to date
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Final
78
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Georgia
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United States of America
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Iowa
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United States of America
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Massachusetts
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United States of America
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Michigan
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United States of America
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Ohio
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United States of America
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Pennsylvania
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Texas
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United States of America
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Utah
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State/province [12]
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Virginia
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United States of America
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Wisconsin
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Country [14]
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Czechia
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Czech Republic
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Finland
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Turku
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France
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Paris
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Hungary
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Budapest
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Italy
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Napoli
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Netherlands
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Amsterdam
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Norway
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Bergen
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Slovenia
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State/province [21]
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Slovenj Gradec
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Spain
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Zaragoza
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Switzerland
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Zürich
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United Kingdom
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Birmingham
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United Kingdom
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Cambridge
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United Kingdom
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London
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United Kingdom
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Salford
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Protalix
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Address
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Country
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Chiesi Farmaceutici S.p.A.
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Address [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
This was a randomized, double-blind, active control study of the enzyme replacement therapy
(ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal
function. Patients who had been treated for approximately 1 year with agalsidase beta and who
had been on a stable dose of that product for at least 6 months were randomized in a 2:1
ratio to either switch to PRX-102 or to continue treatment with agalsidase beta. Both
treatments were delivered by intravenous infusions every two weeks, at a dosage of 1 mg/kg.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02795676
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
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Address
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02795676
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