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

Running, not enrolling · Phase 3

Conditions studied: Fabry Disease

In brief

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.

Key facts

Study ID

NCT03737214

Run by

Idorsia Pharmaceuticals Ltd.

People needed

107

Starts

2018-12-18

Expected to finish

2029-11-01

Last updated by the study team

2026-07-02

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• Fertile male only if agreement 1) to use a condom, 2) to not father a child.

You may not qualify 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.

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 Clinical and Translational Science Institute, UF Clinical Research Center — Gainesville, Florida, United States
• Rush University Medical Center - Dept of Pediatrics — 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
• University of Pennsylvania - Dept of Medicine — Philadelphia, Pennsylvania, United States
• Greenwood Genetics Center — Greenville, South Carolina, United States
• Renal Disease Research Institute LLC — Dallas, Texas, United States
• Baylore University Medical Center — Dallas, Texas, United States
• University of Utah - Division of Medical Genetics, Clinical Genetics Research — Salt Lake City, Utah, United States
• Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States
• Royal Melbourne Hospital - Department of Nephrology — Parkville, Australia
• Royal Perth Hospital, Department of Nephrology — Perth, Australia
• Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse — Vienna, Austria
• University Hospital Ghent (UZ Ghent) — Ghent, Belgium
• University Hospital Gasthuisberg, Leuven (UZ Leuven) — Leuven, Belgium
• University of Calgary - Heritage Medical Research Clinic — Calgary, Canada
• London Health Sciences CTR, Victoria Hospital — London, Canada
• Research Center, Hôpital du Sacré-Coeur de Montréal — Montreal, Canada
• Vancouver General Hospital - Adult Metabolic Diseases Clinic — Vancouver, Canada
• Children's Hospital Research Institute of Manitoba — Winnipeg, Canada
• Raymond Poincaré Hosp - Med Genetics Dept — Garches, France

Full record on ClinicalTrials.gov

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