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A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
NCT05280548 · View on ClinicalTrials.gov ↗
Study Summary
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy. * Study visits will take place approximately every 3 to 6 months * Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.
Conditions Studied
Interventions
- DRUG Venglustat (GZ402671)
- DRUG Agalsidase alfa
- DRUG Agalsidase beta (GZ419828)
- DRUG Migalastat
Study Locations (20)
Other
- Investigational Site Number : 0400001 — Graz
- Investigational Site Number : 1560002 — Beijing
- Investigational Site Number : 1560005 — Beijing
- Investigational Site Number : 1560001 — Chengdu
- Investigational Site Number : 1560007 — Guangzhou
- Investigational Site Number : 1560003 — Shanghai
- Investigational Site Number : 2030001 — Prague
- Investigational Site Number : 2080001 — Copenhagen
Alberta
- Investigational Site Number : 1240003 — Calgary
- Investigational Site Number : 1240006 — Edmonton
Alabama
- University of Alabama -The Kirklin Clinic- Site Number : 8400010 — Birmingham
California
- University of California Los Angeles Medical Center- Site Number : 8400008 — Los Angeles
Georgia
- Emory University School of Medicine - Atlanta- Site Number : 8400009 — Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 — Chicago
New York
- Maryam Banikazemi, MD- Site Number : 8400001 — Hawthorne
Texas
- Renal Disease Research Institute- Site Number : 8400012 — Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 104 participants |
| Start Date | 2022-05-03 |
| Est. Completion | 2027-12-06 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05280548
The ClinicalTrials.gov registry entry for NCT05280548 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 104 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanofi, which has 275 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 4 interventions — of which Venglustat (GZ402671) is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05280548 reports 20 study locations spanning 12 distinct geographic areas — top geographies include Other, Alberta, Alabama. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05280548 about?
NCT05280548 is a clinical study titled "A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease". This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LV...
What is the current status of trial NCT05280548?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 104 participants. The study started on 2022-05-03. Estimated completion is 2027-12-06.
What conditions does trial NCT05280548 study?
This clinical trial studies the following conditions: Fabry Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05280548?
The interventions under investigation include: Venglustat (GZ402671) (DRUG), Agalsidase alfa (DRUG), Agalsidase beta (GZ419828) (DRUG), Migalastat (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05280548?
This trial is sponsored by Sanofi, which has 275 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05280548 being conducted?
This trial has 20 study locations across Alabama, California, Georgia, Illinois, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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