A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
- Registration Number
- NCT03737214
- Lead Sponsor
- Idorsia Pharmaceuticals Ltd.
- Brief 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.
- Detailed Description
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country or until all subjects have reached Month 24 (for subjects participating only in Stage 1) or Month 48 (for subjects participating in Stages 1 and 2) or Month 72 (for subjects participating in Stages 1, 2 and 3), whichever is earliest.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 107
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Lucerastat Lucerastat Dose will be based on subject's eGFR.
- Primary Outcome Measures
Name Time Method Treatment-emergent adverse events (AEs) From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (42)
University of Utah - Division of Medical Genetics, Clinical Genetics Research
🇺🇸Salt Lake City, Utah, United States
University of California Irvine
🇺🇸Irvine, California, United States
University of Pennsylvania - Dept of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
Renal Disease Research Institute LLC
🇺🇸Dallas, Texas, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
🇺🇸Fairfax, Virginia, United States
University Hospital Gasthuisberg, Leuven (UZ Leuven)
🇧🇪Leuven, Belgium
Haukeland University Hospital
🇳🇴Bergen, Norway
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
Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism
🇳🇱Amsterdam, Netherlands
Greenwood Genetics Center
🇺🇸Greenville, South Carolina, United States
Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse
🇦🇹Vienna, Austria
Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital
🇵🇱Kraków, Poland
University Hospital Ghent (UZ Ghent)
🇧🇪Ghent, Belgium
Psychiatrische Universitätsklinik Zürich
🇨🇭Zürich, Switzerland
National Hospital for Neurology and Neurosurgery
🇬🇧London, United Kingdom
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, 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
Baylore University Medical Center
🇺🇸Dallas, Texas, United States
Royal Perth Hospital, Department of Nephrology
🇦🇺Perth, Australia
Royal Melbourne Hospital - Department of Nephrology
🇦🇺Parkville, Australia
University of Calgary - Heritage Medical Research Clinic
🇨🇦Calgary, Canada
Research Center, Hôpital du Sacré-Coeur de Montréal
🇨🇦Montréal, Canada
Vancouver General Hospital - Adult Metabolic Diseases Clinic
🇨🇦Vancouver, Canada
London Health Sciences CTR, Victoria Hospital
🇨🇦London, 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
🇩🇪Hochheim, Germany
Nephrologicum Markgräflerland MVZ GmbH
🇩🇪Müllheim, Germany
Universitätsklinikum Würzburg
🇩🇪Würzburg, Germany
Hospital Universitari de Bellvitge / Nephrology Dpt
🇪🇸Barcelona, Spain
Hospital Quironsalud Zaragoza
🇪🇸Zaragoza, Spain
Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
🇪🇸Madrid, Spain
Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases
🇪🇸Barcelona, Spain
Salford Hospital
🇬🇧Manchester, United Kingdom
Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology
🇬🇧London, United Kingdom
University of Alabama at Birmingham - Nephrology Research Clinic
🇺🇸Birmingham, Alabama, United States
University of Florida Clinical and Translational Science Institute, UF Clinical Research Center
🇺🇸Gainesville, Florida, United States