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Clinical Trials in Canada / NCT03737214
Active, not recruiting Phase 3

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

NCT03737214 · tracked via the Priya Life Science Canada tracker
Phase
Phase 3
Started
2018-12-18
Last updated
2026-08-31

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

Lucerastat

Lucerastat: Administered in hard gelatin capsules containing 250 mg of lucerastat.

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Primary outcome measure(s)

Trial sites (42)

FacilityCityRegionStatus
University of Alabama at Birmingham - Nephrology Research Clinic Birmingham Alabama
University of California Irvine Irvine California
UCSF Benioff Children's Hospital Oakland Oakland California
University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville Florida
Rush University Medical Center - Dept of Pediatrics Chicago Illinois
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics Iowa City Iowa
Massachusetts General Hospital Boston Massachusetts
Infusion Associates Grand Rapids Michigan
University of Pennsylvania - Dept of Medicine Philadelphia Pennsylvania
Greenwood Genetics Center Greenville South Carolina
Renal Disease Research Institute LLC Dallas Texas
Baylore University Medical Center Dallas Texas
University of Utah - Division of Medical Genetics, Clinical Genetics Research Salt Lake City Utah
Lysosomal and Rare Disorders Research and Treatment Center, Inc. Fairfax Virginia
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
Universitätsmedizin Berlin - Charité Campus Mitte Berlin Germany
SphinCS GmbH Höchheim Germany
Nephrologicum Markgräflerland MVZ GmbH Müllheim Germany
Universitätsklinikum Würzburg Würzburg Germany
Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism Amsterdam Netherlands
Haukeland University Hospital Bergen Norway
Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital Krakow Poland
Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy Warsaw Poland
The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases Warsaw Poland
Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases Barcelona Spain
Hospital Universitari de Bellvitge / Nephrology Dpt Barcelona Spain
Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna Madrid Spain
Hospital Quironsalud Zaragoza Zaragoza Spain
Psychiatrische Universitätsklinik Zürich Zurich Switzerland
Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology London United Kingdom

+ 2 more sites — see the full list on the official registry below.

More Idorsia Pharmaceuticals Ltd. trials in Canada

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03737214 on ClinicalTrials.gov ↗ ← All trials in Canada