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Recruiting Phase 3

A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease

NCT07778667 · tracked via the Priya Life Science UK tracker
Phase
Phase 3
Started
2026-12-01
Last updated
2026-11-03

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

Lucerastat →

Lucerastat: Hard gelatine capsules of 250 mg lucerastat

Study summary

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

Eligibility

Sex
MALE
Min age
18 Years
Max age
60 Years
Healthy volunteers
No
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 treatment with an approved therapy or any investigational therapy for Fabry disease for at least 6 months prior to screening. Applicable only to United Kingdom: Potential participants must have been informed of the availability of licensed treatment options for FD and that lucerastat is an unapproved investigational medicinal product that has not yet been assessed by any regulatory authority and determined to have a benefit-risk balance sufficient to support marketing authorization. * 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: Applicable only to Canada and United Kingdom: severe depression or suicidal ideation at screening or history of suicide attempt or behavior within 6 months prior to screening visit. Applicable only to Europe and United States: Any of the following: 1. moderate depression (defined as BDI-II score \> 20 and ≤ 28) if the participant does not agree to a consultation before Visit 2 with an appropriately qualified mental health professional or 2. severe depression (i.e., Beck Depression Inventory-II \[BDI-II\] score \> 28) or 3. suicidal ideation at screening or history of suicide attempt or behavior within 12 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.

Primary outcome measure(s)

  • Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)). — Baseline and Month 18
    Barisoni Lipid Inclusion Scoring System (BLISS) is a quantitative scoring methodology for determining the number of GB3 inclusions in PTCs. A higher BLISS score is indicative of more severe disease on the histologic level.

Trial sites (16)

FacilityCityRegionStatus
University of Alabama of Birmingham Birmingham Alabama Not Yet Recruiting
UCSF Benioff Childens Hospital Oakland California Not Yet Recruiting
UF Health Center for Pediatric Neuromuscular and Rare Diseases Gainesville Florida Not Yet Recruiting
Emory University School of Medicine Atlanta Georgia Not Yet Recruiting
Ann & Robert H. Lurie Childrens Hospital of Chicago Chicago Illinois Not Yet Recruiting
The University of Minnesota, Fairview Health Minneapolis Minnesota Not Yet Recruiting
Hospital of University Pennsylvania, Translational Medicine and Human Genetics Philadelphia Pennsylvania Not Yet Recruiting
Dallas Nephrology Associates (DNA) - Dallas Landry Office Practice Dallas Texas Recruiting
VG Site pharmacy Dept 6 N-279 Halifax Nova Scotia Not Yet Recruiting
CIUSSS-NIM/Hopital du sacre-Coeur de MontrealUnite de Recherche, Local C2030 Montreal Quebec Not Yet Recruiting
Centre Hospitalier Universitaire de Toulouse -Hôpital Purpan Toulouse Midi-Pyrénées Not Yet Recruiting
Hôpital Raymond-Poincaré Garches Île-de-France Region Not Yet Recruiting
Universitaetsklinikum Wuerzburg AöR Grombuehl Bavaria Not Yet Recruiting
Charite Universitaetsmedizin Berlin KöR Mitte State of Berlin Not Yet Recruiting
The Royal Free London NHS Foundation Trust - The Royal Free London City of London Not Yet Recruiting
Salford Royal NHS Foundation Trust part of the Northern Care Salford Lancashire Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07778667 on ClinicalTrials.gov ↗ ← All trials in the UK