Can a new pill stop fatty buildup in fabry disease?
NCT ID NCT07778667
First seen Aug 21, 2026 · Last updated Aug 21, 2026
Summary
This trial tests whether the drug lucerastat can reduce the buildup of a fatty substance called Gb3 in the kidneys of adult men with Fabry disease who have not received prior treatment. Participants will take lucerastat daily for 18 months and undergo kidney biopsies to measure changes. The study aims to see if lucerastat can help manage this genetic condition.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- lucerastat
- What this could lead to
- If successful, lucerastat could become a new treatment option for Fabry disease, potentially slowing kidney damage.
- What could go wrong
- This is a small, early-stage trial (16 participants) without a placebo group, so results may not be conclusive or generalizable. Possible side effects are not yet fully known.
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
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 16 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Mar 2029
An estimate. End dates often move.
- Lead sponsor
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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
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18 to 60 years
- Sex
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Male participants only
- Healthy volunteers
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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 Hide 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: * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a One-Time gene therapy fix fabry disease for years?
- Gene Therapy's lasting promise: can one infusion safely control fabry disease for years?
- Can early enzyme therapy save kidneys in fabry disease?
- Can continued lucerastat access help fabry patients?
- Can a single gene infusion rewrite the story of fabry disease?
- Fabry disease sperm study halted early