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A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Sponsor: Idorsia Pharmaceuticals Ltd.
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.
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
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
107
Start Date
2018-12-18
Completion Date
2029-11
Last Updated
2025-10-21
Healthy Volunteers
No
Conditions
Interventions
Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Locations (42)
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 Perth Hospital, Department of Nephrology
Perth, Perth, Australia
Royal Melbourne Hospital - Department of Nephrology
Parkville, 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
National Hospital for Neurology and Neurosurgery
London, United Kingdom
Salford Hospital
Manchester, United Kingdom