A Phase 3 interventional study of Lucerastat in Fabry Disease, sponsored by Idorsia Pharmaceuticals Ltd.. Completed at 42 sites in 13 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-11-02.
Sponsored by Idorsia Pharmaceuticals Ltd. · Phase 3, Interventional, and Treatment
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest.
Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
244 studies on the registry are indexed under Fabry Disease; 55 are open to participants now.
This study's enrollment of 107 is above the median of 22 across 108 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Idorsia Pharmaceuticals Ltd. is the lead sponsor of 103 studies on the registry; 4 are open to participants now.
Of its 29 completed or terminated interventional studies of FDA-regulated products, 9 (31%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Dose will be based on subject's eGFR.
Drug: Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Treatment-emergent adverse events (AEs)
Time frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
Treatment-emergent serious adverse events (SAEs)
Time frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
Subject estimated glomerular filtration rate (eGFR) slope
Time frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Change in left ventricular mass index (LVMI)
Time frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Change in plasma globotriaosylceramide (Gb3)
Time frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Idorsia Pharmaceuticals Ltd. →