for people ages 18 years and up (full criteria)
at Irvine, California and other locations
study started
completion around



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

Official Title

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease


Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country or until all subjects have reached Month 24 (for subjects participating only in Stage 1) or Month 48 (for subjects participating in Stages 1 and 2) or Month 72 (for subjects participating in Stages 1, 2 and 3), whichever is earliest.


Fabry Disease, Lucerastat


You can join if…

Open to people ages 18 years and up

  • Signed ICF prior to any study-mandated procedure;
  • Subject completed the 6-month, double-blind treatment period in study ID 069A301
  • Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests , 3) not to donate ova.
  • Fertile male only if agreement 1) to use a condom, 2) to not father a child, 3) not to donate sperm.

You CAN'T join if...

  • Pregnant / planning to be become pregnant or lactating subject;
  • Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
  • Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.

    In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:

  • Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
  • Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
  • Subject experienced an event of stroke CTCAE grade 3 or above;
  • Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.


  • University of California Irvine
    Irvine California 92697 United States
  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States


in progress, not accepting new patients
Start Date
Completion Date
Idorsia Pharmaceuticals Ltd.
Phase 3 Fabry Disease Research Study
Study Type
Expecting 107 study participants
Last Updated