A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: ID-069A3022024-513884-20-002018-002210-12
• Study Type: interventional
• Target: 107
• Locations: 12 countries
• Sites: 41

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.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Treatment-emergent adverse events (AEs)

  From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up

Other Outcomes (4)

• 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)

• Change in left ventricular mass index (LVMI)

  From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)

Study Arms (1)

Lucerastat

EXPERIMENTAL

Dose will be based on subject's eGFR.

Drug: Lucerastat

Interventions

Lucerastat DRUG

Administered in hard gelatin capsules containing 250 mg of lucerastat.

Lucerastat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Idorsia Pharmaceuticals Ltd.: lead

Study Sites (42)

University of Alabama at Birmingham - Nephrology Research Clinic

Birmingham, Alabama, 35294, United States

Location

University of California Irvine

Irvine, California, 92697, United States

Location

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

University of Florida Clinical and Translational Science Institute, UF Clinical Research Center

Gainesville, Florida, 32610, United States

Location

Rush University Medical Center - Dept of Pediatrics

Chicago, Illinois, 60612, United States

Location

University of Iowa Stead Family Children's Hospital - Division of Medical Genetics

Iowa City, Iowa, 52242, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Infusion Associates

Grand Rapids, Michigan, 49525, United States

Location

University of Pennsylvania - Dept of Medicine

Philadelphia, Pennsylvania, 19104, United States

Location

Greenwood Genetics Center

Greenville, South Carolina, 29605, United States

Location

Renal Disease Research Institute LLC

Dallas, Texas, 75204, United States

Location

Baylore University Medical Center

Dallas, Texas, 75246, United States

Location

University of Utah - Division of Medical Genetics, Clinical Genetics Research

Salt Lake City, Utah, 84108, United States

Location

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, 22030, United States

Location

Royal Perth Hospital, Department of Nephrology

Perth, Perth, 6000, Australia

Location

Royal Melbourne Hospital - Department of Nephrology

Parkville, 3050, Australia

Location

Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse

Vienna, 1090, Austria

Location

University Hospital Ghent (UZ Ghent)

Ghent, 9000, Belgium

Location

University Hospital Gasthuisberg, Leuven (UZ Leuven)

Leuven, 3000, Belgium

Location

University of Calgary - Heritage Medical Research Clinic

Calgary, T2N 4Z6, Canada

Location

London Health Sciences CTR, Victoria Hospital

London, N6A 5W9, Canada

Location

Research Center, Hôpital du Sacré-Coeur de Montréal

Montreal, H4J 1C5, Canada

Location

Vancouver General Hospital - Adult Metabolic Diseases Clinic

Vancouver, V5Z 1M9, Canada

Location

Children's Hospital Research Institute of Manitoba

Winnipeg, R3E 3P4, Canada

Location

Raymond Poincaré Hosp - Med Genetics Dept

Garches, 92380, France

Location

Universitätsmedizin Berlin - Charité Campus Mitte

Berlin, 10117, Germany

Location

SphinCS GmbH

Höchheim, 65239, Germany

Location

Nephrologicum Markgräflerland MVZ GmbH

Müllheim, 79379, Germany

Location

Universitätsklinikum Würzburg

Würzburg, 97080, Germany

Location

Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism

Amsterdam, 1105 AZ, Netherlands

Location

Haukeland University Hospital

Bergen, 5021, Norway

Location

Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital

Krakow, 31-066, Poland

Location

Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy

Warsaw, 04-628, Poland

Location

The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases

Warsaw, 04-730, Poland

Location

Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases

Barcelona, 08035, Spain

Location

Hospital Universitari de Bellvitge / Nephrology Dpt

Barcelona, 08907, Spain

Location

Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna

Madrid, 28034, Spain

Location

Hospital Quironsalud Zaragoza

Zaragoza, 50012, Spain

Location

Psychiatrische Universitätsklinik Zürich

Zurich, 8032, Switzerland

Location

Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology

London, NW3 2QG, United Kingdom

Location

National Hospital for Neurology and Neurosurgery

London, WC1N 3BG, United Kingdom

Location

Salford Hospital

Manchester, M6 8HD, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 35782623 DERIVED

MeSH Terms

Conditions

Fabry Disease

Interventions

migalastat

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Clinical Trials

  Idorsia Pharmaceuticals Ltd.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multi-center, open-label, uncontrolled, single-arm, extension study

Study Record Dates

• First Submitted: November 6, 2018
• First Posted: November 9, 2018
• Study Start: December 18, 2018
• Primary Completion (Estimated): August 1, 2029
• Study Completion (Estimated): November 1, 2029
• Last Updated: October 21, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2); NL (1); NO (1); ES (4); CH (1); DE (4); PL (3); BE (2); FR (1); GB (3); US (14); CA (5)