A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Public ClinicalTrials.gov record NCT07778667. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 identification
- NCT ID
- NCT07778667
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 16 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 60 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 1, 2026 (Estimated)
- Primary completion
- Feb 13, 2029 (Estimated)
- Completion
- Mar 15, 2029 (Estimated)
- Last update posted
- Nov 3, 2026
2026 – 2029
United States locations
- U.S. sites
- 8
- U.S. states
- 8
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama of Birmingham | Birmingham | Alabama | 35294 | Not yet recruiting |
| UCSF Benioff Childens Hospital | Oakland | California | 94609 | Not yet recruiting |
| UF Health Center for Pediatric Neuromuscular and Rare Diseases | Gainesville | Florida | 32608 | Not yet recruiting |
| Emory University School of Medicine | Atlanta | Georgia | 30322-1013 | Not yet recruiting |
| Ann & Robert H. Lurie Childrens Hospital of Chicago | Chicago | Illinois | 60611 | Not yet recruiting |
| The University of Minnesota, Fairview Health | Minneapolis | Minnesota | 55454 | Not yet recruiting |
| Hospital of University Pennsylvania, Translational Medicine and Human Genetics | Philadelphia | Pennsylvania | 19104 | Not yet recruiting |
| Dallas Nephrology Associates (DNA) - Dallas Landry Office Practice | Dallas | Texas | 75204 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 8 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07778667, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 3, 2026 · Synced Nov 3, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT07778667 live on ClinicalTrials.gov.