Fabry DiseasePhase 3Fab-KlearNCT07778667
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
A Phase 3 study testing Lucerastat in people with Fabry Disease, run by Idorsia Pharmaceuticals Ltd..
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 60 years, men only
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Lucerastat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- No sites listed yet
- Study dates
- Sep 2026to Mar 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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
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:
Read more
- 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
- Conditions
- Fabry Disease
- Being tested
- Lucerastat
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LucerastatDrug
Hard gelatine capsules of 250 mg lucerastat
| Study group | Role | What they receive |
|---|---|---|
| Lucerastat | Experimental | Participants will receive lucerastat (250 mg up to 1000 mg) twice daily (b.i.d). Dose will be determined for each participant based on their estimated glomerular filtration rate (eGFR). Receives: Drug: Lucerastat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).
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.
Measured: Baseline and Month 18
Secondary outcomes (1)
Change from baseline to Month 18 in plasma Gb3 concentration.
Measured: Baseline and Month 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionFeb 2029 (estimated)
- Study completionMar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Multicenter, open-label, single-arm, baseline-controlled study
- Masking
- None (open label)
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Idorsia Pharmaceuticals Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07778667
- Sponsor's protocol ID
- ID-069A306
- Other IDs
- 2026-525207-27-00
- Acronym
- Fab-Klear
- Status last verified
- Aug 2026
- First submitted
- 18 Aug 2026
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07778667
- Acronym / sponsor ID
- Fab-Klear / ID-069A306
- Conditions
- Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.Industry (pharma, biotech, devices)
- Study start
- Sep 2026 (estimated)
- Primary completion
- Feb 2029 (estimated)
- Study completion
- Mar 2029 (estimated)
- First posted
- 21 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multicenter, open-label, single-arm, baseline-controlled study
| Arm | Type | Receives |
|---|---|---|
| Lucerastat | Experimental | Drug: LucerastatParticipants will receive lucerastat (250 mg up to 1000 mg) twice daily (b.i.d). Dose will be determined for each participant based on their estimated glomerular filtration rate (eGFR). |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Confirmed diagnosis of Fabry disease:
- History of at least one of the following clinical manifestations of Fabry disease:
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LucerastatHard gelatine capsules of 250 mg lucerastat | Drug | Lucerastat |
Primary outcome
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)).
Time frame: 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.
1 secondary outcome
Change from baseline to Month 18 in plasma Gb3 concentration.
Time frame: Baseline and Month 18
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Clinical Trials study director
Idorsia Pharmaceuticals Ltd.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lucerastat in people with Fabry Disease, run by Idorsia Pharmaceuticals Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Information USA (+1 856 661 37 21, idorsiaclinicaltrials@idorsia.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline to Month 18 in kidney Gb3 BLISS score (average number of Gb3 inclusions per kidney peritubular capillary (PTC)). (time frame: Baseline and Month 18). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2026.