Phase 3 study · Idorsia · NCT07778667
Idorsia's phase 3 lucerastat study for Fabry disease
About 16 men ages 18 to 60 are planned, and every participant takes lucerastat capsules twice a day for 18 months. There is no placebo, and each participant has a kidney biopsy at the start and at the end of the study.
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Hear when Idorsia's phase 3 lucerastat study opens near you
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Check the main requirements privately
These questions restate the study's public listing in plain words. Your answers stay on this device and are never sent. You can print a one-page summary to go over with your doctor.
1. Are you male and between 18 and 60 years old?
2. Has your Fabry disease been confirmed by an enzyme blood test or a GLA gene test?
The listing asks for very low alpha-galactosidase A enzyme activity, or a disease-causing GLA gene variant with low enzyme activity. Your doctor or genetics clinic can tell you both of these results from your records.3. Have you ever had Fabry nerve pain, whorl-shaped changes in the cornea of the eye, or small dark red skin spots called angiokeratomas?
The eye changes are called cornea verticillata and are usually seen in an eye exam. Your eye doctor's or skin doctor's notes may mention them.4. Have you gone without any approved or study treatment for Fabry disease for at least the last 6 months?
The listing also counts people treated in the past if that treatment ended at least 6 months before screening. Approved Fabry treatments include enzyme replacement infusions and migalastat, a capsule taken by mouth.5. Is your eGFR, the kidney filtering number on your blood test, 45 or higher?
The study measures this itself at screening and uses it to set the lucerastat dose. Your doctor's office can tell you your latest eGFR from a recent blood test.6. Have you had a stroke, mini-stroke, heart attack, unstable angina, heart surgery, or a heart stent or angioplasty in the past 6 months?
7. Have you ever had a gene therapy or a cell therapy, for Fabry disease or anything else?
The study team also checks for these, which are easier to go over with your doctor:
- A blood level of globotriaosylsphingosine (lyso-Gb3), a Fabry marker, of at least 20ng/mL, measured by the study's central lab
- Anything that makes a kidney biopsy unsafe, such as a bleeding disorder, a low platelet count or blood thinners that cannot safely be paused
- Urine albumin over 300mg/g, unless it is already treated with a medicine such as an ACE inhibitor, ARB or SGLT2 inhibitor
- Acute kidney injury in the past 12 months, hemoglobin under 9g/dL, or poorly controlled diabetes with an HbA1c over 8%
- Severe heart failure (NYHA class IV) or a heart failure hospital stay in the past 3 months, or a new heart device such as a pacemaker or a hospital stay for a heart rhythm problem in the past 6 weeks
- Amiodarone, hydroxychloroquine or similar medicines in the past 6 months, since they may affect how the kidney biopsy is read
Sites and contact
US sites from the public ClinicalTrials.gov record, refreshed every hour. Only the study team decides who can join.
- University of Alabama of BirminghamBirmingham, Alabama · Not yet recruiting
- UCSF Benioff Childens HospitalOakland, California · Not yet recruiting
- UF Health Center for Pediatric Neuromuscular and Rare DiseasesGainesville, Florida · Not yet recruiting
- Emory University School of MedicineAtlanta, Georgia · Not yet recruiting
- Ann & Robert H. Lurie Childrens Hospital of ChicagoChicago, Illinois · Not yet recruiting
- The University of Minnesota, Fairview HealthMinneapolis, Minnesota · Not yet recruiting
- Hospital of University Pennsylvania, Translational Medicine and Human GeneticsPhiladelphia, Pennsylvania · Not yet recruiting
- Dallas Nephrology Associates (DNA) - Dallas Landry Office PracticeDallas, Texas · Recruiting
Every site's own contact and the full eligibility list are on the official ClinicalTrials.gov listing for NCT07778667.
Questions people ask about Idorsia's phase 3 lucerastat study
Is Idorsia's phase 3 lucerastat study enrolling now?
ClinicalTrials.gov lists it as recruiting, as of its last update there on 2026-11-03. Only the study team can confirm whether a particular site is still taking people.
Where are the US sites for Idorsia's phase 3 lucerastat study?
The listing names 8 US sites, in Alabama, California, Florida, Georgia, Illinois, Minnesota and 2 other states. The full list is in the sites section of this page.
Who can join Idorsia's phase 3 lucerastat study?
About 16 men ages 18 to 60 are planned, and every participant takes lucerastat capsules twice a day for 18 months. The main listed requirements are in the pre-check on this page, starting with: adult males 18 to 60 years old. Only the study team decides who can join.
How do I contact the Idorsia's phase 3 lucerastat study team?
The listing names this contact: Clinical Trial Information USA, +1 856 661 37 21, idorsiaclinicaltrials@idorsia.com. Each site may also list its own contact on ClinicalTrials.gov.
Is Trial Friend part of this study?
No. Trial Friend is independent of this study and its sponsor, Idorsia. This page restates the public listing in plain words, and nothing here is medical advice; talk with your doctor about whether a study is right for you.
From the public ClinicalTrials.gov record NCT07778667, last updated there 2026-11-03. Questions checked against the listing on November 10, 2026. Trial Friend is independent of this study and its sponsor, and is not a medical provider. Nothing here is medical advice; talk with your doctor about whether a study is right for you.