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Study on Long-Term Safety of Oral Lucerastat for Adults with Fabry Disease

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on studying the long-term safety and tolerability of a medication called Lucerastat in adults with Fabry disease. Fabry disease is a rare genetic disorder that affects the body's ability to break down a certain type of fat, leading to a buildup that can cause a variety of symptoms, including pain, kidney problems, and heart issues. Lucerastat, also known by its code name ACT-434964, is taken orally in the form of a hard capsule.

The purpose of this study is to observe how well patients tolerate Lucerastat over an extended period. Participants in the study will take Lucerastat daily, with a maximum dose of 2000 milligrams per day, for up to 72 weeks. Throughout the study, researchers will monitor participants for any side effects or adverse reactions to the medication. This study is open-label, meaning that both the participants and the researchers know that Lucerastat is being administered, and it is a single-arm study, which means all participants receive the same treatment without a comparison group.

By participating in this study, researchers aim to gather valuable information on the long-term effects of Lucerastat in managing Fabry disease. This information could help in understanding the potential benefits and risks of using Lucerastat as a treatment option for individuals living with this condition. The study is designed to ensure that participants' health and safety are closely monitored throughout the trial period.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after signing and dating the informed consent form (ICF). This document confirms understanding and agreement to participate in the study.

    Eligibility requires completion of a previous 6-month, double-blind treatment period in study ID 069A301.

  2. Step 2

    Medication administration

    The study involves taking Lucerastat, which is a medication in the form of a hard capsule.

    The medication is administered orally. Specific dosage and frequency are determined by the study protocol and communicated by the study team.

  3. Step 3

    Monitoring and assessments

    Regular monitoring is conducted to assess the long-term safety and tolerability of Lucerastat.

    Assessments focus on identifying any treatment-emergent adverse events (AEs) and serious adverse events (SAEs).

  4. Step 4

    Study duration

    The study is an extension study, continuing until the estimated end date of November 23, 2027.

    Participation involves ongoing assessments and medication administration throughout the study period.

Who can join the trial?

5 criteria

  • The patient must have **Fabry disease**, which is a rare genetic condition that affects the body's ability to break down a certain type of fat.
  • The patient must have signed and dated an **Informed Consent Form (ICF)** before any procedures required by the study. This form explains the study and confirms the patient's agreement to participate.
  • The patient must have completed a previous 6-month treatment period in a related study identified as **study ID 069A301**. This means they have already participated in a specific part of the research.
  • The study is open to both **male and female** patients.
  • The study includes patients from certain age groups, specifically those who are **adults** and **older adults**.
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Investigated drugs

Lucerastat is an oral medication being studied for its long-term safety and tolerability in adults with Fabry disease. Fabry disease is a genetic disorder that affects the body's ability to break down a specific type of fat, leading to a buildup that can cause various symptoms. Lucerastat is designed to help manage these symptoms by potentially reducing the accumulation of this fat in the body. This study aims to observe how well patients tolerate the medication over an extended period.

What is already known about the treatment

Lucerastat – Lucerastat is administered orally and is currently being studied in clinical trials to assess its long-term safety and tolerability for treating Fabry disease. It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for lucerastat is Fabry disease, a rare genetic disorder. At the molecular level, lucerastat works by inhibiting the synthesis of certain lipids that accumulate in the cells of individuals with Fabry disease. It is classified pharmacologically as a substrate reduction therapy.

Investigated diseases

Fabry Disease – Fabry disease is a rare genetic disorder caused by the buildup of a specific type of fat in the body's cells. This buildup occurs due to a deficiency of an enzyme called alpha-galactosidase A. The disease primarily affects the skin, kidneys, heart, and nervous system. Symptoms often begin in childhood or adolescence and can include pain, especially in the hands and feet, as well as skin rashes. Over time, the accumulation of fat can lead to more serious complications affecting various organs. The progression of the disease varies among individuals, with some experiencing more severe symptoms than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513884-20-00Protocol codeID-069A302Estimated enrolment103 patientsSponsorIdorsia Pharmaceuticals Ltd.

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