Lucerastat for Fabry Disease
(Fab-Klear Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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
Who Is on the Research Team?
Clinical Trials
Principal Investigator
Idorsia Pharmaceuticals Ltd.
Are You a Good Fit for This Trial?
This trial is for adult men with Fabry disease who have not received treatment in the past 6 months. Participants must show signs of Fabry disease, such as nerve pain or skin changes, and have certain lab results showing low enzyme levels and good kidney function.Inclusion Criteria
What Are the Treatments Tested in This Trial?
Interventions
- Lucerastat
Trial Overview
The study tests lucerastat taken daily for 18 months to see if it lowers a fatty substance (Gb3) that builds up in the kidneys of untreated men with Fabry disease. All participants receive lucerastat and know what they are taking.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
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).
Find a Clinic Near You
Who Is Running the Clinical Trial?
Idorsia Pharmaceuticals Ltd.
Lead Sponsor
Antonio Olivieri
Idorsia Pharmaceuticals Ltd.
Chief Medical Officer since 2024
Not specified
André C. Muller
Idorsia Pharmaceuticals Ltd.
Chief Executive Officer
Not specified
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