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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-513884-20-00 | EU Trial (CTIS) Number | ||
| 2018-002210-12 | EudraCT Number |
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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.
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest.
Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lucerastat | Experimental | Dose will be based on subject's eGFR. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lucerastat | Drug | Administered in hard gelatin capsules containing 250 mg of lucerastat. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment-emergent adverse events (AEs) | From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up |
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| Measure | Description | Time Frame |
|---|---|---|
| Treatment-emergent serious adverse events (SAEs) | From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up | |
| Subject estimated glomerular filtration rate (eGFR) slope | From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) |
Inclusion Criteria:
Exclusion Criteria:
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:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Idorsia Pharmaceuticals Ltd. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham - Nephrology Research Clinic | Birmingham | Alabama | 35294 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35782623 | Derived | Wanner C, Kimonis V, Politei J, Warnock DG, Uceyler N, Frey A, Cornelisse P, Hughes D. Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study. Mol Genet Metab Rep. 2022 Mar 26;31:100862. doi: 10.1016/j.ymgmr.2022.100862. eCollection 2022 Jun. |
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Multi-center, open-label, uncontrolled, single-arm, extension study
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| Change in left ventricular mass index (LVMI) | From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) |
| Change in plasma globotriaosylceramide (Gb3) | From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) |
| University of California Irvine |
| Irvine |
| California |
| 92697 |
| United States |
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | United States |
| University of Florida Clinical and Translational Science Institute, UF Clinical Research Center | Gainesville | Florida | 32610 | United States |
| Rush University Medical Center - Dept of Pediatrics | Chicago | Illinois | 60612 | United States |
| University of Iowa Stead Family Children's Hospital - Division of Medical Genetics | Iowa City | Iowa | 52242 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Infusion Associates | Grand Rapids | Michigan | 49525 | United States |
| University of Pennsylvania - Dept of Medicine | Philadelphia | Pennsylvania | 19104 | United States |
| Greenwood Genetics Center | Greenville | South Carolina | 29605 | United States |
| Renal Disease Research Institute LLC | Dallas | Texas | 75204 | United States |
| Baylore University Medical Center | Dallas | Texas | 75246 | United States |
| University of Utah - Division of Medical Genetics, Clinical Genetics Research | Salt Lake City | Utah | 84108 | United States |
| Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax | Virginia | 22030 | United States |
| Royal Melbourne Hospital - Department of Nephrology | Parkville | 3050 | Australia |
| Royal Perth Hospital, Department of Nephrology | Perth | 6000 | Australia |
| Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse | Vienna | 1090 | Austria |
| University Hospital Ghent (UZ Ghent) | Ghent | 9000 | Belgium |
| University Hospital Gasthuisberg, Leuven (UZ Leuven) | Leuven | 3000 | Belgium |
| University of Calgary - Heritage Medical Research Clinic | Calgary | T2N 4Z6 | Canada |
| London Health Sciences CTR, Victoria Hospital | London | N6A 5W9 | Canada |
| Research Center, Hôpital du Sacré-Coeur de Montréal | Montreal | H4J 1C5 | Canada |
| Vancouver General Hospital - Adult Metabolic Diseases Clinic | Vancouver | V5Z 1M9 | Canada |
| Children's Hospital Research Institute of Manitoba | Winnipeg | R3E 3P4 | Canada |
| Raymond Poincaré Hosp - Med Genetics Dept | Garches | 92380 | France |
| Universitätsmedizin Berlin - Charité Campus Mitte | Berlin | 10117 | Germany |
| SphinCS GmbH | Höchheim | 65239 | Germany |
| Nephrologicum Markgräflerland MVZ GmbH | Müllheim | 79379 | Germany |
| Universitätsklinikum Würzburg | Würzburg | 97080 | Germany |
| Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism | Amsterdam | 1105 AZ | Netherlands |
| Haukeland University Hospital | Bergen | 5021 | Norway |
| Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital | Krakow | 31-066 | Poland |
| Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy | Warsaw | 04-628 | Poland |
| The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases | Warsaw | 04-730 | Poland |
| Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases | Barcelona | 08035 | Spain |
| Hospital Universitari de Bellvitge / Nephrology Dpt | Barcelona | 08907 | Spain |
| Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna | Madrid | 28034 | Spain |
| Hospital Quironsalud Zaragoza | Zaragoza | 50012 | Spain |
| Psychiatrische Universitätsklinik Zürich | Zurich | 8032 | Switzerland |
| Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology | London | NW3 2QG | United Kingdom |
| National Hospital for Neurology and Neurosurgery | London | WC1N 3BG | United Kingdom |
| Salford Hospital | Manchester | M6 8HD | United Kingdom |
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
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| ID | Term |
|---|---|
| C090092 | migalastat |
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