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

NCT07778667 | Not Yet Recruiting Phase 3 FDA Drug

• 2 other identifiers: ID-069A3062026-525207-27-00
• Study Type: interventional
• Target: 16
• Locations: 0 countries
• Sites: N/A

Brief 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

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline and Month 18

Secondary Outcomes (1)

• Change from baseline to Month 18 in plasma Gb3 concentration.

  Baseline and Month 18

Study Arms (1)

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).

Drug: Lucerastat

Interventions

Lucerastat DRUG

Hard gelatine capsules of 250 mg lucerastat

Lucerastat

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Idorsia Pharmaceuticals Ltd.: lead

MeSH Terms

Conditions

Fabry Disease

Interventions

migalastat

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Clinical Trials

  Idorsia Pharmaceuticals Ltd.

  STUDY DIRECTOR

Central Study Contacts

Clinical Trial Information USA

CONTACT

+1 856 661 37 21; idorsiaclinicaltrials@idorsia.com

Clinical Trial Information Europe

CONTACT

+41 58 844 1977; idorsiaclinicaltrials@idorsia.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Multicenter, open-label, single-arm, baseline-controlled study

Study Record Dates

• First Submitted: August 18, 2026
• First Posted: August 21, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): February 1, 2029
• Study Completion (Estimated): March 1, 2029
• Last Updated: August 21, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share