NCT03425539CompletedPhase 3
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
Idorsia Pharmaceuticals Ltd.49 sites in 14 countries118 target enrollmentStarted: June 21, 2018Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Idorsia Pharmaceuticals Ltd.
- Enrollment
- 118
- Locations
- 49
- Primary Endpoint
- Neuropathic Pain Monthly Score: Change From Baseline to Month 6
Study Overview
Brief Summary
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Detailed Description
The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
Lucerastat
Experimental
Intervention: Lucerastat (Drug)
Placebo
Placebo Comparator
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Neuropathic Pain Monthly Score: Change From Baseline to Month 6
Time Frame: From baseline to Month 6 (duration: 6 months)
Neuropathic pain on the modified BPI-SF3: subjects rated their neuropathic pain intensity ("neuropathic pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no neuropathic pain) to 10 (worst imaginable neuropathic pain).
Secondary Outcomes
- Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6(From baseline to Month 6 (duration: 6 months))
- Abdominal Pain Monthly Score: Change From Baseline to Month 6(From baseline to Month 6 (duration: 6 months))
- Number of Days With Diarrhea: Change From Baseline to Month 6(From baseline to Month 6 (duration: 6 months))
Investigators
Study Sites (49)
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