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New oral drug shows promise for fabry disease in Long-Term trial

NCT ID NCT03737214

Disease control Sponsor: Idorsia Pharmaceuticals Ltd. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 01, 2026 · Updated 3 times

Summary

This study looks at the long-term safety of a daily pill called lucerastat for adults with Fabry disease, a rare genetic disorder. About 107 people who completed a previous study will take the drug and be monitored for side effects over several years. The goal is to see if lucerastat is safe to use for ongoing disease control.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 107 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2018

Expected to finish

Nov 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease

Contacts and locations

Locations

  • Baylore University Medical Center

    Dallas, Texas, 75246, United States

  • Children's Hospital Research Institute of Manitoba

    Winnipeg, R3E 3P4, Canada

  • Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital

    Krakow, 31-066, Poland

  • Greenwood Genetics Center

    Greenville, South Carolina, 29605, United States

  • Haukeland University Hospital

    Bergen, 5021, Norway

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

    Amsterdam, 1105 AZ, Netherlands

  • Hospital Quironsalud Zaragoza

    Zaragoza, 50012, Spain

  • Hospital Universitari de Bellvitge / Nephrology Dpt

    Barcelona, 08907, Spain

  • Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna

    Madrid, 28034, Spain

  • Infusion Associates

    Grand Rapids, Michigan, 49525, United States

  • London Health Sciences CTR, Victoria Hospital

    London, N6A 5W9, Canada

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

    Fairfax, Virginia, 22030, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

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

    Vienna, 1090, Austria

  • Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy

    Warsaw, 04-628, Poland

  • National Hospital for Neurology and Neurosurgery

    London, WC1N 3BG, United Kingdom

  • Nephrologicum Markgräflerland MVZ GmbH

    Müllheim, 79379, Germany

  • Psychiatrische Universitätsklinik Zürich

    Zurich, 8032, Switzerland

  • Raymond Poincaré Hosp - Med Genetics Dept

    Garches, 92380, France

  • Renal Disease Research Institute LLC

    Dallas, Texas, 75204, United States

  • Research Center, Hôpital du Sacré-Coeur de Montréal

    Montreal, H4J 1C5, Canada

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

    London, NW3 2QG, United Kingdom

  • Royal Melbourne Hospital - Department of Nephrology

    Parkville, 3050, Australia

  • Royal Perth Hospital, Department of Nephrology

    Perth, 6000, Australia

  • Rush University Medical Center - Dept of Pediatrics

    Chicago, Illinois, 60612, United States

  • Salford Hospital

    Manchester, M6 8HD, United Kingdom

  • SphinCS GmbH

    Höchheim, 65239, Germany

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

    Warsaw, 04-730, Poland

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • University Hospital Gasthuisberg, Leuven (UZ Leuven)

    Leuven, 3000, Belgium

  • University Hospital Ghent (UZ Ghent)

    Ghent, 9000, Belgium

  • University of Alabama at Birmingham - Nephrology Research Clinic

    Birmingham, Alabama, 35294, United States

  • University of Calgary - Heritage Medical Research Clinic

    Calgary, T2N 4Z6, Canada

  • University of California Irvine

    Irvine, California, 92697, United States

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

    Gainesville, Florida, 32610, United States

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

    Iowa City, Iowa, 52242, United States

  • University of Pennsylvania - Dept of Medicine

    Philadelphia, Pennsylvania, 19104, United States

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

    Salt Lake City, Utah, 84108, United States

  • Universitätsklinikum Würzburg

    Würzburg, 97080, Germany

  • Universitätsmedizin Berlin - Charité Campus Mitte

    Berlin, 10117, Germany

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

    Barcelona, 08035, Spain

  • Vancouver General Hospital - Adult Metabolic Diseases Clinic

    Vancouver, V5Z 1M9, Canada

More trials for these conditions

Other studies related to the condition(s) this trial covers.