Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
- Registration Number
- NCT03566017
- Lead Sponsor
- Chiesi Farmaceutici S.p.A.
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 97
- Completion of study PB-102-F20, PB-102-F03, or PB-102-F30
- The patient signs informed consent
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.
Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Experimental open label pegunigalsidase alfa pegunigalsidase alfa
- Primary Outcome Measures
Name Time Method Evaluation of treatment-related adverse events Throughout the study, 7 years CTCAE v4.03
- Secondary Outcome Measures
Name Time Method Kidney function 1 Every 6 months throughout trial, 7 years Estimated glomerular filtration rate (eGFRCKD-EPI)
Cardiac assessment Every 12 months to end of the study, 7 years Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)
Biomarkers for Fabry disease Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months. plasma Lyso-Gb3 and Gb3
Record of pain medication use Every two weeks for 7 years Frequency of pain medication use, or pre-infusion medication
Kidney function 2 Every 6 months to the end of the study, 7 years Protein/Creatinine ratio, spot urine test (UPCR)
Pain assessment Every 6 months up to the end of the study, 7 years short form Brief Pain Inventory (BPI)
Symptom assessment Every 12 months up to the end of the study, 7 years Mainz Severity Score Index (MSSI)
Quality of life assessment Every 6 months up to the end of the study, 7 years quality of life (EQ-5D-5L)
Trial Locations
- Locations (30)
General Hospital Slovenj Gradec
🇸🇮Slovenj Gradec, Slovenia
Infusion Associates
🇺🇸Grand Rapids, Michigan, United States
Institute of Metabolic Disease
🇺🇸Dallas, Texas, United States
University of Utah Hospitals & Clinics
🇺🇸Salt Lake City, Utah, United States
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
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Renal Disease Research Institute, LLC - Dallas
🇺🇸Dallas, Texas, United States
O+O Alpan LLC
🇺🇸Fairfax, Virginia, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Royal Melbourne Hospital
🇦🇺Parkville, Victoria, Australia
Capital District Health Authority
🇨🇦Halifax, Nova Scotia, Canada
Vseobecna fakultni nemocnice v Praze
🇨🇿Prague, Czech Republic, Czechia
Turku University Central Hospital
🇫🇮Turku, Finland
Hospital Raymond-Poincaré
🇫🇷Garches, France
Semmelweis Egyetem
🇭🇺Budapest, Hungary
Azienda Ospedaliera Universitaria "Federico II"
🇮🇹Napoli, Via Pansini, Italy
Academisch Medisch Centrum
🇳🇱Amsterdam, Netherlands
Haukeland University Hospital Klinisk Forskningspost
🇳🇴Bergen, Norway
Hospital de Dia Quiron Zaragoza
🇪🇸Zaragoza, Spain
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
🇬🇧Edgbaston, Birmingham, United Kingdom
Salford Royal
🇬🇧Salford, Greater Manchester, United Kingdom
Addenbrooke's Hospital
🇬🇧Cambridge, United Kingdom
The Royal Free Hospital
🇬🇧London, United Kingdom