EUCTR2017-003369-85-ESActive, not recruitingPhase 1
A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Idorsia Pharmaceuticals Ltd
- Enrollment
- 108
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Signed and dated ICF prior to any study-mandated procedure.
- •2. Male or female subjects; 18-years old and above.
- •3. FD diagnosis confirmed with local genetic test results (i.e., presence of at least 1 mutation in GLA, the gene coding for alfa-
- •galactosidase A).
- •4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 97
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 11
Exclusion Criteria
- •1. Pregnant, planning to be become pregnant up to 30 days after study treatment discontinuation or lactating subject;
- •2. Severe renal insufficiency defined as an estimated glomerular filtration rate (eGFR) per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 30 mL/min/1.73 m2 at screening;
- •3. Subject on regular dialysis for the treatment of chronic kidney disease;
- •4. Subject has undergone, or is on a waiting list for, or is scheduled to undergo kidney or other organ transplantation;
- •5. Known and documented transient ischemic attack, stroke, unstable angina or myocardial infarction within 6 months prior to screening;
- •6. Clinically significant unstable cardiac disease in the opinion of the investigator (e.g., uncontrolled symptomatic arrhythmia, New York Heart Association class III or IV congestive heart failure);
- •7. Any other subject at high risk for developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
- •8. Subject planned for imminent initiation of treatment with ERT.
Investigators
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