Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Completed · Phase 3 · Has a placebo group
Conditions studied: Fabry Disease
In brief
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Key facts
- Study ID
- NCT03425539
- Run by
- Idorsia Pharmaceuticals Ltd.
- People needed
- 118
- Starts
- 2018-06-21
- Expected to finish
- 2021-09-02
- Last updated by the study team
- 2024-08-09
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Signed and dated ICF prior to any study-mandated procedure;
- Male or female adult subjects;
- FD diagnosis confirmed with local genetic test results;
- Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
- Enzyme replacement therapy (ERT) status:
- Subject never treated with ERT; or
- Subject has not received ERT for at least 6 months prior to screening; or
- 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.
- A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
- Subjects with moderate or severe neuropathic pain during the screening period.
You may not qualify if…
- Pregnant, planning to be become pregnant, or lactating subject.
- Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
- Subject on regular dialysis for the treatment of chronic kidney disease.
- Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
- Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.
Where it is running
- University of Alabama at Birmingham - Nephrology Research Clinic — Birmingham, Alabama, United States
- University of California Irvine — Irvine, California, United States
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
- University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation — Gainesville, Florida, United States
- Emory University School of Medicine; Department of Human Genetics — Atlanta, Georgia, United States
- Rush University Medical Center — Chicago, Illinois, United States
- University of Iowa Stead Family Children's Hospital - Division of Medical Genetics — Iowa City, Iowa, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Infusion Associates — Grand Rapids, Michigan, United States
- Duke University Medical Center — Durham, North Carolina, United States
- Oregon Health and Science University — Portland, Oregon, United States
- University of Pennsylvania — Philadelphia, Pennsylvania, United States
- Children's Hospital of Pittsburgh (UPMC) — Pittsburgh, Pennsylvania, United States
- Greenwood Genetic Center — Greenville, South Carolina, United States
- Research Baylor Institute of Metabolic Disease — Dallas, Texas, United States
- University of Utah - Division of Medical Genetics — Salt Lake City, Utah, United States
- Lysosomal and Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States
- Royal Melbourne Hospital - Department of Nephrology — Parkville, Australia
- Royal Perth Hospital, Department of Nephrology — Perth, Australia
- Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse — Vienna, Austria
- University Hospital Ghent — Ghent, Belgium
- University Hospital Leuven — Leuven, Belgium
- London Health Sciences Centre - Victoria Hospital — London, Ontario, Canada
- M.A.G.I.C Clinic Ltd — Calgary, Canada
- Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology — Halifax, Canada
Full record on ClinicalTrials.gov
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