Fabry Disease clinical trials at UC Irvine

• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

  Sorry, in progress, not accepting new patients

  A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

  Irvine, California and other locations

• Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease

  Sorry, accepting new patients by invitation only

  The objective of 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-F20 or PB-102-F30.

  Orange, California and other locations

• Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

  Sorry, in progress, not accepting new patients

  This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with 1mg/kg agalsidase beta or to treatment with 1 mg/kg of PRX-102. The identity of the enzyme will be blinded to the patient and the investigator. Patients will receive intravenous infusions every two weeks. Patients will be randomized in a 2:1 ratio of PRX-102 to agalsidase beta. Randomization will be stratified by urinary protein to creatinine ratio (UPCR) of < or ≥ 1 g/g by spot urine sample. No more than 50% of the patients will be female.

  Orange, California and other locations

Last updated: November 2018