Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
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|ClinicalTrials.gov Identifier: NCT03018730|
Recruitment Status : Completed
First Posted : January 12, 2017
Results First Posted : June 29, 2022
Last Update Posted : August 26, 2022
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Biological: PRX-102 (pegunigalsidase alfa)||Phase 3|
Dosage and administration details:
pegunigalsidase alfa individual dose for each patient was prepared according to the patient's weight. Pegunigalsidase alfa administrated at 1 mg/kg, intravenously over 3 hours, every 2 weeks. After the first 2 months of treatment with pegunigalsidase alfa, infusion time may be reduced gradually to 1.5 hours pending patient tolerability.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||22 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Actual Study Start Date :||May 17, 2017|
|Actual Primary Completion Date :||December 17, 2019|
|Actual Study Completion Date :||January 9, 2020|
PRX-102 infusion every 2 weeks
Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
- Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03 [ Time Frame: 12 months ]Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment
- eGFR [ Time Frame: Baseline and Month 12 (week 52) ]eGFR was calculated based on the serum creatinine values that were assessed at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 according to the CKD-EPI formula, baseline and Month 12 (week 52) reported. The absolute change in eGFR from baseline measurement at visit 1 prior to first PRX-102 infusion to last measurement at Month 12 was summarized using descriptive statistics.
- Mean Annualised Change in eGFR (Slope) [ Time Frame: Pre-switch, Post-switch ]
The annualized change in eGFR (slope) per patient was calculated with all available eGFR values using a linear regression.
The mean pre-switch slope is the eGFR slope during screening period and pre-infusion visit 1 (while on Replagal®).
The mean post-switch slope is the eGFR slope during PRX-102 treatment, calculated based on eGFR vales at weeks 4, 8, 12, 16, 20, 26, 30, 34, 38, 42, 46, 52 after visit 1.
The mean change in eGFR slope from pre- to post-switch is the mean difference between the two slopes.
eGFR was calculated based on the serum creatinine values according to the CKD-EPI formula.
- Plasma Lyso-Gb3 [ Time Frame: Baseline and month 12 (week 52) ]Plasma Lyso-Gb3 is Fabry disease specific biomarker that can assess treatment outcome which was measured at Baseline and weeks 12, 26, 38, 52. Baseline and Month 12 (week 52) reported.
- Number of Participants According to Protein/Creatinine Ratio (UPCR) [ Time Frame: 12 months ]Urine Protein to Creatinine Ratio (UPCR), assessed by spot urine test, at Month 12 (Week 52).Number of Participants According to Protein/Creatinine Ratio (UPCR) level
- Left Ventricular Mass Index (g/m^2) by MRI [ Time Frame: 12 months ]Left ventricular mass was determined based on cardiac MRI data and the LVMI was indexed to patient's body surface area (g/m^2). In male patients the normal range for LVMI was 57-91 g/m^2, in female patients 47-77 g/m^2.
- Quality of Life EQ VAS [ Time Frame: 12 months ]The EQ VAS, of the EQ 5D 5L questionnaire, records the subject's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score "100") and 'Worst imaginable health state' (score "0").
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
|Royal Melbourne Hospital|
|Parkville, Victoria, Australia, 3050|
|Canada, Nova Scotia|
|Halifax, Nova Scotia, Canada, B3H 1V8|
|Vseobecna fakultni nemocnice v Praze|
|Academisch Medisch Centrum|
|Helse Bergen HF Haukeland Universitetssykehus|
|General Hospital Slovenj Gradec|
|Slovenj Gradec, Slovenia, SI-2380|
|Queen Elizabeth Hospital, Department of Neurology,|
|Edgbaston, Birmingham, United Kingdom, B15 2TH|
|The Royal Free Hospital|
|London, United Kingdom, NW3 2QG|
|Salford Royal NHS Foundation Trust|
|Salford, United Kingdom, M6 8HD|
|Study Director:||Raul Chertkoff, MD||Protalix Ltd.|