- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03737214
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
January 10, 2024 updated by: Idorsia Pharmaceuticals Ltd.
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
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease
Study Overview
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.
Study Type
Interventional
Enrollment (Estimated)
107
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Parkville, Australia, 3050
- Royal Melbourne Hospital - Department of Nephrology
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Perth, Australia, 6000
- Royal Perth Hospital, Department of Nephrology
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Vienna, Austria, 1090
- Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse
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Ghent, Belgium, 9000
- University Hospital Ghent (UZ Ghent)
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Leuven, Belgium, 3000
- University Hospital Gasthuisberg, Leuven (UZ Leuven)
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Calgary, Canada, T2N 4Z6
- University of Calgary - Heritage Medical Research Clinic
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London, Canada, N6A 5W9
- London Health Sciences CTR, Victoria Hospital
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Montréal, Canada, H4J 1C5
- Research Center, Hôpital du Sacré-Coeur de Montréal
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Vancouver, Canada, V5Z 1M9
- Vancouver General Hospital - Adult Metabolic Diseases Clinic
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Winnipeg, Canada, R3E 3P4
- Children's Hospital Research Institute of Manitoba
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Garches, France, 92380
- Raymond Poincaré Hosp - Med Genetics Dept
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Berlin, Germany, 10117
- Universitätsmedizin Berlin - Charité Campus Mitte
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Hochheim, Germany, 65239
- SphinCS GmbH
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Müllheim, Germany, 79379
- Nephrologicum Markgräflerland MVZ GmbH
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Würzburg, Germany, 97080
- Universitätsklinikum Würzburg
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Amsterdam, Netherlands, 1105 AZ
- Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism
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Bergen, Norway, 5021
- Haukeland University Hospital
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Kraków, Poland, 31-066
- Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital
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Warsaw, Poland, 04-628
- Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy
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Warsaw, Poland, 04-730
- The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases
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Barcelona, Spain, 08035
- Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases
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Barcelona, Spain, 08907
- Hospital Universitari de Bellvitge / Nephrology Dpt
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Madrid, Spain, 28034
- Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
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Zaragoza, Spain, 50012
- Hospital Quironsalud Zaragoza
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Zürich, Switzerland, 8032
- Psychiatrische Universitätsklinik Zürich
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London, United Kingdom, WC1N 3BG
- National Hospital For Neurology and Neurosurgery
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London, United Kingdom, NW3 2QG
- Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology
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Manchester, United Kingdom, M6 8HD
- Salford Hospital
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Alabama
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Birmingham, Alabama, United States, 35294
- University of Alabama at Birmingham - Nephrology Research Clinic
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California
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Irvine, California, United States, 92697
- University of California Irvine
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Oakland, California, United States, 94609
- UCSF Benioff Children's Hospital Oakland
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Florida
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Gainesville, Florida, United States, 32610
- University of Florida Clinical and Translational Science Institute, UF Clinical Research Center
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Illinois
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Chicago, Illinois, United States, 60612
- Rush University Medical Center - Dept of Pediatrics
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
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Massachusetts
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Boston, Massachusetts, United States, 02114
- Massachusetts General Hospital
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Michigan
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Grand Rapids, Michigan, United States, 49525
- Infusion Associates
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- University of Pennsylvania - Dept of Medicine
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South Carolina
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Greenville, South Carolina, United States, 29605
- Greenwood Genetics Center
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Texas
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Dallas, Texas, United States, 75204
- Renal Disease Research Institute LLC
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Dallas, Texas, United States, 75246
- Baylore University Medical Center
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Utah
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Salt Lake City, Utah, United States, 84108
- University of Utah - Division of Medical Genetics, Clinical Genetics Research
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Virginia
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Fairfax, Virginia, United States, 22030
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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 , 3) not to donate ova.
- Fertile male only if agreement 1) to use a condom, 2) to not father a child, 3) not to donate sperm.
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Lucerastat
Dose will be based on subject's eGFR.
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Administered in hard gelatin capsules containing 250 mg of lucerastat.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Treatment-emergent adverse events (AEs)
Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
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From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
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Other Outcome Measures
Outcome Measure |
Time Frame |
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Treatment-emergent serious adverse events (SAEs)
Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
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From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
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Subject estimated glomerular filtration rate (eGFR) slope
Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
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From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
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Change in left ventricular mass index (LVMI)
Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
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From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
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Change in plasma globotriaosylceramide (Gb3)
Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
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From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Clinical Trials, Idorsia Pharmaceuticals Ltd.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 18, 2018
Primary Completion (Estimated)
October 29, 2025
Study Completion (Estimated)
November 29, 2025
Study Registration Dates
First Submitted
November 6, 2018
First Submitted That Met QC Criteria
November 7, 2018
First Posted (Actual)
November 9, 2018
Study Record Updates
Last Update Posted (Estimated)
January 11, 2024
Last Update Submitted That Met QC Criteria
January 10, 2024
Last Verified
January 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Metabolic Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Cerebral Small Vessel Diseases
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Fabry Disease
Other Study ID Numbers
- ID-069A302
- 2018-002210-12 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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CENTOGENE GmbH RostockCompletedFabry Disease | Anderson-Fabry Disease | Fabry´s DiseaseArgentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
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Wuerzburg University HospitalTakedaEnrolling by invitationLysosomal Storage Diseases | Fabry Disease | Fabry Disease, Cardiac Variant | HCM - Hypertrophic Cardiomyopathy | Anderson Fabry DiseaseGermany
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University Hospital, RouenUnknownAnderson-Fabry DiseaseFrance
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Sangamo TherapeuticsEnrolling by invitationFabry Disease | Fabry Disease, Cardiac VariantUnited States, Australia, United Kingdom
-
Amicus Therapeutics France SASActive, not recruitingFabry Disease | Anderson Fabry DiseaseFrance
-
Academisch Medisch Centrum - Universiteit van Amsterdam...RecruitingFabry Disease | Fabry Disease, Cardiac VariantNetherlands
-
Taipei Veterans General Hospital, TaiwanSanofiUnknownFabry Disease, Cardiac Variant
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University of CambridgeSanofiRecruiting
-
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-
Wolfson Medical CenterUnknownFabry Disease in the Young StrokeIsrael
Clinical Trials on Lucerastat
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Idorsia Pharmaceuticals Ltd.CompletedFabry DiseaseUnited States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
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Idorsia Pharmaceuticals Ltd.Completed
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ActelionCompletedHealthy SubjectsUnited Kingdom
-
ActelionCompletedHealthy SubjectsUnited Kingdom
-
Idorsia Pharmaceuticals Ltd.Completed
-
Idorsia Pharmaceuticals Ltd.CompletedHealthy SubjectsGermany