ClinicalTrials.gov record NCT07778667
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
A Multicenter, Open-label, Single-arm, Baseline-controlled Trial to Assess the Efficacy and Safety of Lucerastat in Treatment-naïve/Pseudo-naïve Adult Male Participants With Fabry Disease
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label, single-arm Phase 3 trial studies lucerastat in adult men with Fabry disease who have not received Fabry-specific treatment in at least the past 6 months. Participants take oral lucerastat twice daily for 18 months and are followed for up to 21 months. The study includes kidney biopsies at the start and Month 18, about 10 clinic visits, and blood testing. The main outcomes are change in kidney Gb3 inclusions and plasma Gb3; safety is also assessed.
What it is studying. Whether treatment with lucerastat changes the amount of globotriaosylceramide (Gb3) in the kidney and what its safety profile is in untreated adult male participants with Fabry disease.
What the researchers hope to learn. Scientifically, the trial aims to measure change from baseline to Month 18 in kidney Gb3 using the BLISS score (average number of Gb3 inclusions per kidney peritubular capillary), change in plasma Gb3 concentration, and to assess the safety of lucerastat in this population.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The purpose of this clinical trial is to learn how well lucerastat works and how safe it is in untreated adult male participants with Fabry disease. The main question this clinical trial aims to answer is:
- Does treatment with lucerastat affects the amount of globotriaosylceramide (Gb3), a fatty substance that builds up in the kidneys, in untreated adult men with Fabry disease?
This is an open-label, single-arm trial, which means that participants will know which trial medication they receive and only one trial medication will be given. Trial participants will:
- Take lucerastat every day for 18 months
- Have kidney biopsies at the end and start of the trial
- Visit the clinic 10 times for check-up and tests
- Take part in the trial for up to 21 months in total
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Lucerastat
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Confirmed diagnosis of Fabry disease:
- Plasma and/or leukocyte α-galactosidase A (α-GalA) < 1% mean normal levels or
- Known "pathogenic" or "likely pathogenic" Gene coding for α-galactosidase A (GLA) variant with a low level (i.e., < 30% mean normal levels) of plasma and/or leukocyte α-GalA.
- History of at least one of the following clinical manifestations of Fabry disease:
- Neuropathic pain
- Cornea verticillata
- Angiokeratoma
- Treatment-naïve or pseudo-naïve i.e. without prior treatment with an approved or any investigational therapy for Fabry disease within at least 6 months prior to screening.
- Plasma globotriaosylsphingosine ≥ 20 ng/ml (as assessed centrally).
- Screening eGFR (central laboratory) ≥ 45 mL/min/1.73 m2.
Exclusion criteria
- Any intercurrent condition or concomitant therapy considered a contraindication for kidney biopsy, as per local standard of care, or in the investigator's opinion may preclude accurate interpretation of trial data.
- Urine albumin-to-creatinine ratio > 300 mg/g at screening (central laboratory) unless treated with background therapy, such as Angiotensin-converting enzyme inhibitors, Angiotensin receptor blocker or Sodium-glucose cotransporter 2 inhibitors, as per local practice.
- Inherited or acquired coagulopathy, uncorrected bleeding disorders, international normalized ratio > 1.5, platelet count < 50,000/μL or inability to safely hold anticoagulants or antiplatelet therapy as applicable per local practice (usually 1-2 days for anticoagulants and 3-7 days for antiplatelets).
- Hemoglobin level < 9.0 g/dL at screening.
- History of acute kidney injury within 12 months prior to screening visit.
- Documented poorly controlled diabetes mellitus (i.e., Hemoglobin A1c > 8.0% at screening as reported by the central laboratory).
- History of cerebrovascular event (e.g. stroke, transient ischemic attack), cardiovascular event (e.g., myocardial infarction, unstable angina), cardiac surgery (e.g., coronary artery bypass graft, valvular repair/replacement) or percutaneous coronary intervention within 6 months prior to screening.
- Congestive heart failure New York Heart Association class IV or hospitalization for heart failure within 3 months prior to screening.
- Implementation of cardiac device (e.g., pacemaker, implantable cardioverter defibrillator, cardiac resynchronization therapy device) or hospitalization for arrhythmia within 6 weeks prior to screening.
- Any other known factor or disease that might interfere with treatment compliance, trial conduct, or interpretation of the results, such as drug or alcohol dependence or psychiatric disease including severe depression or suicidal ideation at screening or history of suicide attempt or behavior within 6 months prior to screening visit.
- Previous exposure to gene or cell therapy.
- Use of cationic amphiphilic drugs, such as amiodarone or hydroxychloroquine that may preclude accurate interpretation of kidney biopsy data within 6 months prior to screening.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Take oral lucerastat capsules twice daily for 18 months, with the dose determined by kidney function (eGFR). Attend about 10 clinic visits for check-ups and tests and be followed for up to 21 months total. Undergo kidney biopsies at the start of the trial and again at Month 18 to assess kidney Gb3. Have routine blood, urine, and other assessments during clinic visits.
How the study is structured. Open-label, single-group (no comparison arm) Phase 3 trial where all participants receive oral lucerastat capsules twice daily for 18 months (dose 250 mg up to 1000 mg b.i.d., determined by eGFR). Key procedures named in the registry: kidney biopsy at baseline and at Month 18, plasma Gb3 measurements, approximately 10 clinic visits for checks and tests, and overall participation up to 21 months.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Fabry disease
Important dates
- Study start
- September 2026 (estimated)
- Primary completion
- February 2029 (estimated)
- Study completion
- March 2029 (estimated)
- Target enrollment
- 16 participants (estimated)
- Last updated on registry
- Aug 21, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Clinical Trial Information USA 📞 +1 856 661 37 21 ✉ idorsiaclinicaltrials@idorsia.com
- Clinical Trial Information Europe 📞 +41 58 844 1977 ✉ idorsiaclinicaltrials@idorsia.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- Treatment-naïve
- Has not previously received the treatment, or the type of treatment, that the study is about.
- prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- eGFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- concomitant therapy
- Other medicines a participant takes during the study. Some must be reported; some are restricted.
- contraindication
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- standard of care
- Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
- hydroxychloroquine
- A medicine used for some autoimmune conditions. It appears in eligibility criteria when a study requires or excludes its use.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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