Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks (BRIGHT)
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|ClinicalTrials.gov Identifier: NCT03180840|
Recruitment Status : Completed
First Posted : June 8, 2017
Results First Posted : January 5, 2023
Last Update Posted : January 5, 2023
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Biological: Pegunigalsidase alfa||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Switch over study in patients previously receiving either agalsidase alfa or agalsidase beta and switched to pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease.|
|Masking:||None (Open Label)|
|Official Title:||Phase 3 Open-Label Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2mg/kg IV Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy Fabrazyme® or Replagal™|
|Actual Study Start Date :||July 10, 2017|
|Actual Primary Completion Date :||August 1, 2020|
|Actual Study Completion Date :||August 1, 2020|
Experimental: Pegunigalsidase alfa
Pegunigalsidase alfa 2 mg/kg intravenous infusion every 4 weeks
Biological: Pegunigalsidase alfa
Pegunigalsidase alfa 2 mg/kg every 4 weeks
- Number of Participants With Treatment-related Adverse Events (TEAE) as Assessed by CTCAE v4.03 [ Time Frame: Month 12 ]Results represent the number of treatment-emergent adverse events (TEAE) that were considered possibly, probably, or definitely related to treatment.
- Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline and Month 12 (week 52) ]eGFR was calculated based on the serum creatinine values that were assessed at Day 1, weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52 according to the CKD-EPI formula, baseline and Month 12 (week 52) reported. The change in eGFR from baseline measurement at Day 1 prior to first PRX-102 infusion to last measurement at Month 12 was summarized using descriptive statistics. The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes.
- Plasma Lyso-Gb3 [ Time Frame: Baseline and month 12 (Week 52) ]
Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker that can assess treatment outcome, for which was measured at Baseline, weeks 12, 24, 40 and 52. The mean Plasma Lyso-Gb3 concentrations at baseline and Month 12 (week 52) and the mean change from Baseline reported.
The change from baseline to month 12 was calculated for each subject and the reported values is the mean (Standard Error) of these changes.
- Quality of Life by EQ-VAS [ Time Frame: Baseline and 12 months (week 52) ]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). The change from baseline to month 12 was calculated for each subject and the reported value is the mean (Standard Error) of these changes.
- Pharmacokinetics - Cmax [ Time Frame: Day 1, Month 9 or 11, and Month 12 ]Pharmacokinetic (PK) parameters were derived from the plasma concentration versus time profiles. Cmax is the maximal plasma concentration of a drug after administration. Results reported represent the values following a single dosing of the study drug.
- Pharmacokinetics - AUC [ Time Frame: Day 1, Month 9 or 11, and Month 12. ]PK parameters were derived from the plasma concentration versus time profiles. AUC is the area under the plasma concentration curve from 0 hour to infinity. Results reported represent the values following a single dosing of the study drug.
- Pharmacokinetics - Terminal Half Life [ Time Frame: Day 1, Month 9 or 11, and Month 12. ]PK parameters were derived from the plasma concentration versus time profiles. t1/2 = half life. Results reported represent the values following a single dosing of the study drug.
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): NCT03180840
|United States, Alabama|
|Birmingham, Alabama, United States, 35294|
|United States, Georgia|
|Emory University School of Medicine|
|Atlanta, Georgia, United States, 30322|
|United States, Iowa|
|University of Iowa Hospitals and Clinics|
|Iowa City, Iowa, United States, 52242|
|United States, Michigan|
|Grand Rapids, Michigan, United States, 49525|
|United States, Texas|
|Institute of Metabolic Disease|
|Dallas, Texas, United States, 75226|
|United States, Utah|
|University of Utah Hospital & Clinics|
|Salt Lake City, Utah, United States, 84132|
|United States, Virginia|
|O & O Alpan|
|Fairfax, Virginia, United States, 22030|
|Edegem, Belgium, 2650|
|Fakultní poliklinika Všeobecné fakultní nemocnice v Praze|
|Praha 2, Czechia, 120 00|
|Copenhagen, Denmark, 2100|
|Azienda Ospedaliera Universitaria "Federico II"|
|Helse Bergen HF Haukeland Universitetssykehus|
|Bergen, Norway, 5021|
|Cambridge, United Kingdom, CB2 0QQ|
|The Royal Free Hospital|
|London, United Kingdom, NW3 2QG|
|Study Director:||Sari Alon||Sr. Director Regulatory Affairs|