Fabry Disease clinical trials at UC Health

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

  open to eligible people ages 18 years and up

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

  at UC Irvine UCLA UCSF

• Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

  open to eligible people ages 18 years and up

  This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.

  at UC Irvine UCLA UCSF

• 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.

  at UC Irvine

• 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.

  at UC Irvine UCLA UCSD

Last updated: June 23, 2020