A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Treatment-emergent adverse events (AEs) — From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
Trial sites (42)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 Spain
Other trials for the same condition
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 Spain