The objective of CLI-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.
This is an open-label study. Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment is until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor. For the analysis, available efficacy and safety parameters will be summarized using descriptive statistics.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
97
Recombinant human alpha galactosidase A
Evaluation of treatment-related adverse events
CTCAE v4.03
Time frame: Throughout the study, 7 years
Kidney function 1
Estimated glomerular filtration rate (eGFRCKD-EPI)
Time frame: Every 6 months throughout trial, 7 years
Cardiac assessment
Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)
Time frame: Every 12 months to end of the study, 7 years
Biomarkers for Fabry disease
plasma Lyso-Gb3 and Gb3
Time frame: Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
Record of pain medication use
Frequency of pain medication use, or pre-infusion medication
Time frame: Every two weeks for 7 years
Kidney function 2
Protein/Creatinine ratio, spot urine test (UPCR)
Time frame: Every 6 months to the end of the study, 7 years
Pain assessment
short form Brief Pain Inventory (BPI)
Time frame: Every 6 months up to the end of the study, 7 years
Symptom assessment
Mainz Severity Score Index (MSSI)
Time frame: Every 12 months up to the end of the study, 7 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
UAB Medicine
Birmingham, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
University of California San Diego
La Jolla, California, United States
University of California Irvine Center
Orange, California, United States
University of Florida
Gainesville, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
Infusion Associates
Grand Rapids, Michigan, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
...and 20 more locations
Quality of life assessment
quality of life (EQ-5D-5L)
Time frame: Every 6 months up to the end of the study, 7 years