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

Sponsor
Idorsia Pharmaceuticals Ltd.
Study ID
NCT07778667
Phase
PHASE3
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
MALE
Age
18 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • Lucerastat — DRUG
    Hard gelatine capsules of 250 mg lucerastat

Study Details

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

Key Dates

First listed
Aug 21, 2026
Start date
Sep 30, 2026
Status verified
Aug 2026
Primary completion
Feb 28, 2029
Completion
Mar 31, 2029

Study Design

Enrollment
16 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Lucerastat
    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).

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 ]

Central Contacts

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