Phase 3
N=118
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Fabry Disease
Bottom Line
View on ClinicalTrials.gov: NCT03425539 ↗Enrolled (actual)
118
Serious AEs
5.1%
Results posted
Aug 2024
Primary outcome: Primary: Neuropathic Pain Monthly Score: Change From Baseline to Month 6 — -1.64; -2.05 score on a scale — p=0.3189
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Lucerastat (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Idorsia Pharmaceuticals Ltd.
- Primary completion
- Aug 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Neuropathic Pain Monthly Score: Change From Baseline to Month 6 |
-1.64; -2.05 | 0.3189 |
| SECONDARY Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6 |
-672.68; 200.84 | <0.0001 sig |
| SECONDARY Abdominal Pain Monthly Score: Change From Baseline to Month 6 |
-1.37; -1.68 | 0.4676 |
| SECONDARY Number of Days With Diarrhea: Change From Baseline to Month 6 |
-3.45; -3.95 | 0.8986 |
Summary
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Eligibility Criteria
Inclusion Criteria
- Signed and dated ICF prior to any study-mandated procedure;
- Male or female adult subjects;
- FD diagnosis confirmed with local genetic test results;
- Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
- Enzyme replacement therapy (ERT) status:
- Subject never treated with ERT; or
- Subject has not received ERT for at least 6 months prior to screening; or
- Subject treated with ERT since at least 12 months at the time of the screening visit, and agreeing to stop ERT for approximately 8 months.
- A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
- Subjects with moderate or severe neuropathic pain during the screening period.
Exclusion Criteria
- Pregnant, planning to be become pregnant, or lactating subject.
- Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
- Subject on regular dialysis for the treatment of chronic kidney disease.
- Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
- Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.
Data sourced from ClinicalTrials.gov (NCT03425539). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.