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

August 27, 2026 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. 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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
107

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Available

Available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 outside the clinical trial. See expanded access record.

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

  • Australia
      • Parkville, Australia, 3050
        • Royal Melbourne Hospital - Department of Nephrology
      • Perth, Australia, 6000
        • Royal Perth Hospital, Department of Nephrology
  • Austria
      • Vienna, Austria, 1090
        • Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse
  • Belgium
      • Ghent, Belgium, 9000
        • University Hospital Ghent (UZ Ghent)
      • Leuven, Belgium, 3000
        • University Hospital Gasthuisberg, Leuven (UZ Leuven)
  • Canada
      • Calgary, Canada, T2N 4Z6
        • University of Calgary - Heritage Medical Research Clinic
      • London, Canada, N6A 5W9
        • London Health Sciences CTR, Victoria Hospital
      • Montreal, Canada, H4J 1C5
        • Research Center, Hôpital du Sacré-Coeur de Montréal
      • Vancouver, Canada, V5Z 1M9
        • Vancouver General Hospital - Adult Metabolic Diseases Clinic
      • Winnipeg, Canada, R3E 3P4
        • Children's Hospital Research Institute of Manitoba
  • France
      • Garches, France, 92380
        • Raymond Poincaré Hosp - Med Genetics Dept
  • Germany
      • Berlin, Germany, 10117
        • Universitätsmedizin Berlin - Charité Campus Mitte
      • Höchheim, Germany, 65239
        • SphinCS GmbH
      • Müllheim, Germany, 79379
        • Nephrologicum Markgräflerland MVZ GmbH
      • Würzburg, Germany, 97080
        • Universitätsklinikum Würzburg
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism
  • Norway
      • Bergen, Norway, 5021
        • Haukeland University Hospital
  • Poland
      • Krakow, Poland, 31-066
        • Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital
      • Warsaw, Poland, 04-628
        • Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy
      • Warsaw, Poland, 04-730
        • The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases
  • Spain
      • Barcelona, Spain, 08035
        • Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases
      • Barcelona, Spain, 08907
        • Hospital Universitari de Bellvitge / Nephrology Dpt
      • Madrid, Spain, 28034
        • Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
      • Zaragoza, Spain, 50012
        • Hospital Quironsalud Zaragoza
  • Switzerland
      • Zurich, Switzerland, 8032
        • Psychiatrische Universitätsklinik Zürich
  • United Kingdom
      • London, United Kingdom, WC1N 3BG
        • National Hospital For Neurology and Neurosurgery
      • London, United Kingdom, NW3 2QG
        • Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology
      • Manchester, United Kingdom, M6 8HD
        • Salford Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama at Birmingham - Nephrology Research Clinic
    • California
      • Irvine, California, United States, 92697
        • University of California Irvine
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital Oakland
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida Clinical and Translational Science Institute, UF Clinical Research Center
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center - Dept of Pediatrics
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Michigan
      • Grand Rapids, Michigan, United States, 49525
        • Infusion Associates
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania - Dept of Medicine
    • South Carolina
      • Greenville, South Carolina, United States, 29605
        • Greenwood Genetics Center
    • Texas
      • Dallas, Texas, United States, 75204
        • Renal Disease Research Institute LLC
      • Dallas, Texas, United States, 75246
        • Baylore University Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • University of Utah - Division of Medical Genetics, Clinical Genetics Research
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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.
  • 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.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Lucerastat
Dose will be based on subject's eGFR.
Drug: Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Treatment-emergent adverse events (AEs)
Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Time Frame
Subject estimated glomerular filtration rate (eGFR) slope
Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Change in left ventricular mass index (LVMI)
Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Change in plasma globotriaosylceramide (Gb3)
Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Treatment-emergent serious adverse events (SAEs)
Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Idorsia Pharmaceuticals Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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.

General Publications

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 18, 2018

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 6, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 7, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 9, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 31, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ID-069A302
  • 2018 (U.S. NIH Grant/Contract)
  • 2018-002210-12 (EudraCT Number)
  • 2024-513884-20-00 (Ctis)

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

product manufactured in and exported from the U.S.

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.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings