The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
29
Recombinant human alpha galactosidase A
UAB Medicine
Birmingham, Alabama, United States
Emory University School of Medicine
Atlanta, Georgia, United States
University of Iowa Hospitals and Clinica
Iowa City, Iowa, United States
Evaluation of treatment-related adverse events
CTCAE v4.03
Time frame: Throughout the study, 364 weeks
Kidney function 1
Estimated glomerular filtration rate (eGFRCKD-EPI)
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Cardiac assessment
Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Time frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Biomarkers for Fabry disease
Plasma Lyso-Gb3 and Gb3
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Kidney function 2
Protein/Creatinine ratio (UPCR), spot urine test
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Clinical assessment
Record of pain medication and pre-medication use
Time frame: Every four weeks throughout the duration of the study, 364 weeks
Pain assessment
Short form Brief Pain Inventory (BPI)
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Symptom assessment
Mainz Severity Score Index (MSSI)
Time frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Infusion Associates
Grand Rapids, Michigan, United States
Renal Disease Research Institute, LLC
Dallas, Texas, United States
University of Utah Hospitals & Clinics
Salt Lake City, Utah, United States
O & O Alpan
Fairfax, Virginia, United States
UZ Antwerpen
Edegem, Belgium
Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
Prague, Czechia
Medical Endocrinology PE 2132, Rigshospitalet
Copenhagen, Denmark
...and 4 more locations
Quality of life assessment
Quality of life (EQ-5D-5L)
Time frame: Every 6 months throughout the duration of the study, 364 weeks