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

NCT07778667 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-08-21

No results posted yet for this study

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

Interventions

DRUG

Lucerastat

Hard gelatine capsules of 250 mg lucerastat

Sponsors & Collaborators

  • Idorsia Pharmaceuticals Ltd.

    lead INDUSTRY

Principal Investigators

  • Clinical Trials · Idorsia Pharmaceuticals Ltd.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
60 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-30
Primary Completion
2029-02-28
Completion
2029-03-31
FDA Drug
Yes

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07778667 on ClinicalTrials.gov