Fabry Disease clinical trials at University of California Health

• Fabry Disease Registry & Pregnancy Sub-registry

  open to all eligible people

  The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Registry are: - To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; - To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; - To characterize and describe the Fabry population as a whole; - To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

  at UCLA

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

  Sorry, accepting new patients by invitation only

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

  at UC Irvine UCSF

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

  Sorry, in progress, not accepting new patients

  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: March 2, 2021