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



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

Official Title

A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease


The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.


Fabry Disease Lucerastat


You can join if…

Open to people ages 18 years and up

  1. Signed and dated ICF prior to any study-mandated procedure;
  2. Male or female adult subjects;
  3. FD diagnosis confirmed with local genetic test results;
  4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
  5. Enzyme replacement therapy (ERT) status:
  6. Subject never treated with ERT; or
  7. Subject has not received ERT for at least 6 months prior to screening; or
  8. Subject treated with ERT since at least 12 months at the time of the screening visit, and agreeing to stop ERT for approximately 8 months.
  9. A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
  10. Subjects with moderate or severe neuropathic pain during the screening period.

You CAN'T join if...

  1. Pregnant, planning to be become pregnant, or lactating subject.
  2. Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
  3. Subject on regular dialysis for the treatment of chronic kidney disease.
  4. Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
  5. Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
  6. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.


  • University of California Irvine
    Irvine California 92696 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
Study Type
Last Updated