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

NCT ID: NCT03737214

Last Updated: 2025-10-21

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 107 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-12-18
• Study Completion Date: 2029-11-30

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 (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.

Conditions

Fabry Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Lucerastat

Dose will be based on subject's eGFR.

Group Type: EXPERIMENTAL

Lucerastat

Intervention Type: DRUG

Administered in hard gelatin capsules containing 250 mg of lucerastat.

Interventions

Lucerastat

Administered in hard gelatin capsules containing 250 mg of lucerastat.

Intervention Type: DRUG

Eligibility Criteria

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.
• Fertile male only if agreement 1) to use a condom, 2) to not father a child.

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Idorsia Pharmaceuticals Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Idorsia Pharmaceuticals Ltd.

Locations

University of Alabama at Birmingham - Nephrology Research Clinic

Birmingham, Alabama, United States

University of California Irvine

Irvine, California, United States

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

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

Gainesville, Florida, 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

University of Pennsylvania - Dept of Medicine

Philadelphia, Pennsylvania, United States

Greenwood Genetics Center

Greenville, South Carolina, United States

Renal Disease Research Institute LLC

Dallas, Texas, United States

Baylore University Medical Center

Dallas, Texas, United States

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

Salt Lake City, Utah, United States

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, United States

Royal Perth Hospital, Department of Nephrology

Perth, Perth, Australia

Royal Melbourne Hospital - Department of Nephrology

Parkville, , 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

National Hospital for Neurology and Neurosurgery

London, , United Kingdom

Salford Hospital

Manchester, , United Kingdom

Countries

United States; Australia; Austria; Belgium; Canada; France; Germany; Netherlands; Norway; Poland; Spain; Switzerland; United Kingdom

References

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.

Reference Type: DERIVED

PMID: 35782623 (View on PubMed)

Other Identifiers

2024-513884-20-00

Identifier Type: CTIS

Identifier Source: secondary_id

2018-002210-12

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ID-069A302

Identifier Type: -

Identifier Source: org_study_id